Two children died following a stabbing in Southport at a Taylor Swift-themed dance event in Stockport yesterday.

Nine more children were injured, with six in critical condition. Two adults were also injured according to Merseyside Police.

The free leaflet, from the University’s Parenting and Families Research Group available here was developed for the 91ֱ�� Arena bombing and then for the Grenfell fire, with trauma experts from around the country.

It is designed to  help those affected through the critical first few days after the  trauma, but also in the months that follow.

The leaflet is  designed to help parents and caregivers cope with their own emotions and stress and  will help them to understand common reactions in children and how best to care for them.

Professor Rachel Calam, who helped develop the leaflet said: “ This is a tragic incident; parents, children and teachers will need good psychological support to help the navigate through the coming days and months.

“What they are going through might include difficulties sleeping, thoughts and memories of what has happened popping into mind, bad dreams, irritability, feeling low, behavioural problems and avoiding activities they used to enjoy.

“This leaflet is advises them how keep going in such difficult times, and that experiencing some distress like that is entirely normal. There is no one way of feeling after a trauma.

“We developed this information to help anyone wondering how best to help their child through such a frightening and upsetting experience. We hope you find it helpful.”

For more For more family advice, visit the NHS  MindEd website .

Download the advice

“A ban would cause young people to find alternatives to existing social media platforms that may be harder for parents, educators, researchers and legislators to study and monitor,” said Dr Eiko Fried, Associate Professor in Clinical Psychology at Leiden University. “Rather than imposing restrictions, efforts should be directed towards educating young people, their guardians and educators on navigating the digital landscape safely, and on regulations which ensure that social media companies design age-appropriate features and algorithms.”

A new report from Child of the North and Anne Longfield’s Centre for Young Lives think tank has set out a plan for the Government to boost children’s mental health through the education system, as half of England’s school children will still be without access to Mental Health Support Teams after 2025 under current plans.

The report, “Improving mental health and wellbeing with and through educational settings”, sets out the crucial role schools can play in supporting children’s mental health and promoting and supporting wellbeing. With children spending more time in school than in any other formal institutional structure, educational settings provide the ideal opportunity to reach large numbers of children simultaneously and can also facilitate intervention with pupils displaying early mental health or behavioural symptoms.

It is the third in a series of Child of the North/Centre for Young Lives reports to be published during 2024, focusing on how both the Government and Opposition can reset their vision for children to put the life chances of young people at the heart of policy making and delivery.

The report comes amid a national epidemic of children’s mental health problems. In 2022, 18% of children aged 7-to-16-years-old and 22% of young people aged 17-to-24 had a probable mental health condition. Despite some extra investment in recent years, the children’s mental health system is blighted by chronic waiting lists and a postcode lottery of provision, and thousands of children and young people continue to struggle without support. Over 32,000 children had been waiting over two years for help at the end of 2022/3. The consequences for school attendance, educational achievement, mental health problems in adulthood, as well as over-stretched public services, economic productivity, and society’s overall wellbeing are enormous.

The report calls on the Government to expand the mental health support offered through schools and educational settings from primary school onwards, without placing extra burdens on teachers.

Its recommendations include harnessing the power of digital technology in a way that benefits the mental health of children by rolling out school-based research surveys like the existing #BeeWell and Age of Wonder projects nationally. This would gather local information about children’s mental health and wellbeing, identify geographical hotspots and determine when the ‘emotional temperature’ of the school is in the danger zone, so that schools can offer early support.

The report also recommends:

· Expanding the mental health support offered through schools and educational settings, starting in the primary school years, to all schools. Mental Health Support Teams (MHSTs) are known to provide effective help to schools, but most schools still do not have access to them. The Government’s current plans mean that from 2025 half of England’s 8 million school age children will still not have access to a MHST in their school, should they need it. The work of MHSTs should be widened so it is not just focused on only one-to-one support for children with moderate-to-severe mental health problems, but is also focussed on peer group support and school-wide prevention strategies, including mental health hubs. This can be achieved by involving the community and voluntary sector, alongside health and social care services.

· Supporting the creation of a network of ‘one stop shop’ local online NHS information hubs, based on NHS Healthier Together, to signpost children and families to appropriate local mental health support where it is available. A ‘one stop shop’ would allow children, families, and schools to learn together about the local mental health support offered in their locality and how it can be accessed. The information hub would allow schools to work together more effectively with parents and children to create a supportive learning environment, tailored to local services and the local community.

· Tackling the upstream determinants of poor mental health, including early support for neurodivergent children. The evidence shows that pre-school and primary school experience can increase the risk for mental health conditions. Government’s strategy to improve the social and emotional wellbeing of young people should include a focus on the pre-school and primary school years. A national strategy to provide greater support for children with neurodiversity in their preschool years to tackle early determinants of poor mental health is also vital.

· Addressing the workforce crisis in educational psychology provision to encourage a larger number of graduate psychologists to support schools, alongside teacher training and career development that equips teaching staff to create classroom and school environments that promote pupil wellbeing and support the mental health needs of pupils. Government could and should mandate the provision of such training in the education and CPD of teaching staff.

To highlight the scale of mental health problems among young people, the report also includes preliminary data gathered from 5,000 children and young people in Bradford that reveals the shocking rise of eating disorders in the area, including:

· One in five (21%) of Year 9 pupils in Bradford reporting a probable eating disorder.

· 18% of 12-to-15-year-olds in Bradford reporting symptoms indicative of a probable eating disorder (the national rate among 11-to-16-year olds is 13%).

· 17% of 12-to-15 year olds reported self-harm in the last 12 months, with a higher prevalence in girls (20%) compared to boys (13%).

The study also highlights two priority issues raised by children and young people in Bradford as detrimental to their mental health - problems with lack of sleep and with loneliness. These findings are seen elsewhere. A recent #BeeWell survey examined the relationship between sleep quality in approximately 35,000 young people in more than 150 schools across Greater 91ֱ��. #BeeWell found that more than four in ten young people reported not getting enough sleep.

Anne Longfield, Executive Chair of the Centre for Young Lives, said:

“The rise in the number of children experiencing mental health problems is an ongoing crisis not only for those children and families experiencing it now, but for our country’s future.

“I have heard so many heartbreaking stories of the lengths children and parents have gone to get support – including, sadly, suicide attempts – but we still seem a long way away from providing the prevention, early help, and treatment that every young person with mental health problems needs.

“As an anchor in children’s lives, schools have a crucial role to play in supporting children’s mental health and wellbeing. Yet half of the school age children in England – four million children – will not have access to Mental Health Support Teams under current plans. We need to rocket-boost support in schools if we hope to bring down the numbers of children who are struggling with mental health problems.

“The current school attendance crisis is likely to be driven in part by children with mental health problems who are unwilling or unable to attend school. We know already that children and young people with mental health conditions are more likely to be absent from school, and that poor mental health significantly impacts on school attendance and outcomes.

“At the next election, the parties will put forward their proposals for improving children’s mental health. Labour has already pledged to recruit more staff, introduce specialist mental health support for children in every school, and deliver an open access children and young people’s mental health hub for every community. But there should be a cross-party ambition to reduce the prevalence of children’s mental health conditions by half over the next 10 years, and all politicians should agree that the current system is failing too many children and needs urgent attention.”

Dr Camilla Kingdon, former President of the Royal College of Paediatrics and Child Health said:

“There is a huge evidence base for the importance of good mental health in childhood. However, sadly nearly 50% of lifetime mental health conditions are established by 14 years. We have a crucial window of opportunity to intervene to support children with mental health problems. We cannot let these children slip through the system without help.

“The UK needs to prioritise mental health and wellbeing of children for the sake of our children - and all our futures. There are solutions at our fingertips - we just need the political will to make it happen.”

Professor Mark Mon Williams, Child of The North report series editor, said:

“There is no better measure of the health of a nation than the mental wellbeing of its children and young people. The statistics on mental health in children are heartbreaking and demand immediate action. The UK must prioritise the mental health and wellbeing of its children and young people if it wants to enjoy long term prosperity. This report shows how the next Government could and should invest in the UK’s future wellbeing.”

Dr Ruth Wadman, Research Fellow for the Age of Wonder Adolescent Mental Health Collaboratory, said:

“Our children and young people need good mental health and wellbeing to develop and flourish. There is an urgent need to step-up our efforts to prevent mental health conditions and to intervene early when they emerge. The report shows that schools can play a key role in promoting good mental health and wellbeing, both by harnessing the power of data and by listening to children and young people.”

• In September 2023, there was a 27% rise in new autism referrals over the last year. 
• In July 2022, more than 125,000 people were waiting for an autism assessment by mental health services, an increase of 34% since the previous October. By July 2023, this number had risen to more than 143,000. Figures published in September 2023, show there were 157,809 patients with an open referral for suspected autism.
• 93% of children did not receive an appointment within 13 weeks of being referred. 
• The number of children yet to receive an appointment after 13 weeks has increased by 36% since Covid-19, and there has been a 21% increase in the last twelve months.  
• More than one in four parents have waited over three years to receive support for their child.
• The evidence shows that children born to mothers without educational qualifications will receive an autism diagnosis two years later than their peers, and that issues around timely identification and support are exacerbated for girls, who are more likely to be misdiagnosed and diagnosed later than boys, or not at all.
• Children and young people from ethnic minority backgrounds are experiencing lower rates of identification of autism and often experience more severe difficulties.

The report warns that the failure to provide the right autism support can lead to poor long-term outcomes for autistic children, including an increased prevalence of connected conditions such as mental ill health and a greater risk of school exclusion or not attending school. Data from the Connected Bradford database included in the report reveals that children who had been referred but were still waiting for an assessment were at greatest risk of being excluded from secondary school. 

Autistic children who had a diagnosis were less likely to be excluded from school, compared to those awaiting an assessment, suggesting a diagnosis and subsequent support has a protective effect. With waiting times increasing, there is a growing risk to education outcomes, with evidence suggesting that many autistic children are ending up in expensive Alternative Provision.

The report also describes how a major barrier to existing systems is the perceived need for a medical diagnosis of autism before any child can receive support, with the perception among schools that this is a requirement, preventing some children from accessing support. Given the long waiting lists, many autistic children are not receiving the support they need because they do not have a formal diagnosis.

It makes three key recommendations to Government which have the potential the decrease the long-term costs associated with not acting early:

• Building effective partnerships between education and health professionals for assessing and supporting autistic children. This should include delivering assessments in education settings and making a holistic offer of support in schools and nurseries before and after a formal diagnosis is made.
• Providing and extending access to mandatory Continuing Professional Development (CPD) courses for health, education, and social care professionals that improve understanding and awareness of autism (and related issues). These courses should include information on how to create “neurodiverse friendly” environments, and particularly raise awareness of autism in girls and ethnic minority groups. Additional training should be co-produced by individuals with lived experience and delivered to professionals and integrated into undergraduate health and education professional training to improve the identification of autistic girls.
• Creating formal partnerships at a local authority level comprising sector leaders (including schools, health, voluntary services, faith, universities, educational psychologists, and businesses) to oversee a prioritised governmental ward-level approach to addressing the autism crisis. The partnership should focus on its most disadvantaged wards and provide leadership in trialing data-driven, community and family co-produced, “whole system” approaches to improve autism support with and through education settings.

“The number of autistic children seeking support is at a record high and the number waiting for an assessment has rocketed since Covid,” said Anne Longfield, Executive Chair of the Centre for Young Lives. “The autism assessment crisis is leaving thousands of children without the support they need and parents having to battle their way through a nightmare process that can take years to resolve.

“The pressure and stress this is putting on families and children can have terrible and damaging consequences for mental health and for children’s education chances. Autistic children with a referral who are waiting for an assessment are at significantly greater risk of exclusion from school, with all the further risks that can bring. If waiting times continue to increase, so can the risk of increased exclusion and poorer educational outcomes for autistic children.

“The evidence shows the need to move to a system of support that responds to the needs of autistic children, rather than waiting for diagnosis before any help appears. Without urgent reform, we cannot hope to improve the life chances of the next generation.”

• Girls report lower wellbeing than boys, and LGBTQ+ young people report significantly lower wellbeing than their cisgender, heterosexual peers.
• Only one in three young people are meeting the Chief Medical Officer’s recommendation of doing one hour of physical activity per day. This drops to one in four girls.
• Over 40% of Year 9 students report that they aren’t getting enough sleep to feel awake and concentrate at school (2022).
• Deeper analysis indicates that, by tackling bullying, we could prevent nearly 1 in 5 cases of young people's significant feelings of worry or sadness.
• Neighbourhood-level data reveals that characteristics such as income disparity, health deprivation, crime risk and more are significantly correlated to different domains or drivers of wellbeing.

These findings are already informing activity across Greater 91ֱ��, with schools, voluntary sector organisations and children’s services working closely with young people to interpret and act on the results. For instance:

• A social prescribing and youth-led commissioning pilot in 5 neighbourhoods in GM used the survey findings to allocate £20,000
• #BeeWell measures are utilised by the Greater 91ֱ�� Combined Authority (GMCA), ensuring young people’s wellbeing will be at the heart of future strategy
• Investment into arts, culture and wellbeing initiatives amongst voluntary sector partners and within schools
• Investment into a pilot project from Greater 91ֱ�� Integrated Care on improving young LGBTQ+ people’s wellbeing
• Voluntary sector partners leading campaigns to improve physical activity amongst girls in response to inequalities identified by #BeeWell data
• Targeted responses to improve physical activity and nutrition by schools

Professor Jessica Deighton, Director of Applied Research and Evaluation, Anna Freud said:  “If we are to take the mental health and wellbeing of our children and young people seriously, then we need to have a robust survey that can authoritatively collate data from English counties and cities. #BeeWell’s impact on measurement in this area of work could help the UK to improve in the PISA rankings internationally. The voices of our young people are one of the most powerful tools that #BeeWell lets us hear.”

James Robertson, #BeeWell National Director, said: “The enthusiasm across Greater 91ֱ�� to deliver the #BeeWell programme has been inspiring. I’m so grateful to our partners at the GMCA, academics at the University of Manchester, and to all the schools who delivered the programme for a third year running. All their hard work amplifies the voices of young people at a time when listening to young people has never been more important. Driving action across the city region in response to the latest survey continues to be front and centre at #BeeWell.”

Councillor Mark Hunter, Greater 91ֱ�� portfolio lead for Children and Young People, and leader of Stockport Council, said: “It is always vital that we listen to our young people across Greater 91ֱ�� and truly understand the unprecedented difficulties they have faced over recent years. I’m delighted that the #BeeWell programme has once again given young people the opportunity to have their voices heard and be involved in something that really does make a meaningful difference. We are absolutely determined to improve all aspects of young peoples’ wellbeing in Greater 91ֱ��."

Five children under one year of age with the condition also known as mucopolysaccharidosis type II (MPS II) will be treated with autologous hematopoietic stem cell (HSC) gene therapy.

The children will continue to receive enzyme replacement therapy during treatment, but once the gene therapy begins to work, the research team say part of the trial aims to remove the need for weekly enzyme replacement therapy over the child’s lifetime, while the other aim is to safely target the brain disease suffered by these patients.

The combined phase 1 and 2 clinical trial initiated by University of Manchester researchers, is now open to recruitment. AVROBIO, the previous funders of the program, have returned the license to The University of Manchester.

The study will be carried out at Royal 91ֱ�� Children’s Hospital (RMCH) in collaboration with the 91ֱ�� Centre for Genomic Medicine at Saint Mary’s Hospital – both part of Manchester University NHS Foundation Trust (MFT), will trial the drug for the treatment for this rare inherited disorder, which was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester.

Professor Bigger and his team this month published a in Molecular Therapy clinical Methods which validates the proof-of-concept outcomes findings in mice, providing further long-term efficacy data.

The trial aims to recruit up to five patients with severe MPS II who are aged between 3 months and 12 months at time of consent. Inclusion criteria are for children in the above age range with a confirmed diagnosis of severe MPSII who may already be on enzyme replacement therapy but have not yet developmentally declined.

It will be a 24-month, single-arm, open label study which will evaluate the HSC gene therapy’s safety and tolerability, as well as its pharmacodynamic and clinical efficacy.

Children with severe Hunter syndrome cannot properly break down complex sugar molecules and have widespread symptoms including rapid and progressive learning and memory problems, heart and lung dysfunction, hyperactivity and behavioural problems, bone and joint malformations and hearing impairment.

The UK Medicines and Healthcare Products Regulatory Agency (MHRA), Research Ethics Committee (REC), and Health Research Authority (HRA) have all approved the clinical trial application that was submitted by The University of Manchester in August 2022.

The clinical trial will be led by Professor Rob Wynn, Consultant Paediatric Haematologist at RMCH, together with Professor Simon Jones, Consultant in Paediatric Inherited Metabolic Disease at Saint Mary’s Hospital, and Professor Bigger at The University of Manchester. The University of Manchester will act as trial sponsor.

Children with Hunter syndrome have a missing gene, meaning they cannot produce an important enzyme called iduronate-2-sulfatase or IDS. The gene therapy works by collecting HSCs from the patient and inserting a working copy of the gene into the HSCs using a lentiviral gene therapy vector. The modified HSCs are then infused back into the patient to engraft in the bone marrow. Following successful engraftment of modified HSCs in the bone marrow, these cells start to produce daughter blood cells which contain the IDS gene and enzyme which are distributed throughout the body, including the brain.

 Professor Bigger said: “This is a next generation stem cell gene therapy approach, which allows transit of the IDS enzyme into the brain. The newly inserted IDS gene produces an IDS enzyme that contains a proprietary ApoEII-tagged sequence, which can bind to ApoE-dependent receptors on the blood brain barrier, and move enzyme into the brain more efficiently, thus potentially normalizing brain pathology.

“This should speed up delivery of enzyme to the brain, where it is most needed as we can leverage all the enzyme produced by the blood to do this rather than just relying on the engraftment of monocyte cells from the blood into the brain.”

He added: “We’re very excited by the pre-clinical studies we carried out in mice, which showed the potential to correct disease in the body and normalize brain pathology.

“Mice with Hunter syndrome treated with the HSC gene therapy showed dramatic improvement in their condition, including normalization of working memory problems, and skeletal features such as the cheekbone dimensions and the width of the humerus and femur bones.”

The trial is the culmination of a more than 15-year collaborative effort with Professor Wynn and Professor Jones at MFT to develop HSC gene therapies for neurological lysosomal disorders and is now the second potential neurological gene therapy that this collaborative team has brought into the clinical setting.

Bob Stevens, chief executive of the MPS society said: “This ground-breaking trial initiated by The University of Manchester offers the possibility of new treatment options in the future for patients with the severest form of MPSII Hunters. We look forward to hearing the outcome of this trial, with cautious optimism and hope that science will offer the chance of a ‘Rare Life Lived Better’.”

“This study shows that even small changes in what fathers do, and in how schools and early years settings engage with parents, can have a lasting impact on children's learning,” said Andrew Gwynne MP, Chair of the All-Party Parliamentary Group on Fatherhood. “It's absolutely crucial that that fathers aren't treated as an afterthought.”

"#BeeWell gives a voice to young people and highlights important issues that we’re facing in Greater 91ֱ�� like the previous survey finding that shows only 9 in every average classroom are getting enough sleep to concentrate in school," said Maria, a member of the #BeeWell Youth Steering Group. “I’m really looking forward to sharing my voice as part of this year's survey and encourage all year 10 students to do the same so that #BeeWell can act with us to create change.”

“We are excited to be working with partners to launch the third annual #BeeWell survey, especially this year as the programme is building on the success in Greater 91ֱ�� by expanding into Hampshire, Isle of Wight, Portsmouth and Southampton,” said Professor Jessica Deighton from The Anna Freud Centre. “We really look forward to hearing the voices of more young people across the country to understand more about what supports their wellbeing.” 

“It is crucial that we hear directly from the young people themselves in conversations about their wellbeing,” said David Gregson from The Gregson Family Foundation. “I’m delighted that such a high proportion of schools have committed to taking part in the #BeeWell survey this autumn. The feedback young people share, will give weight to the debate about their wellbeing, informing how we collectively act to deliver improvements.” 

For more information about the #BeeWell programme visit https://beewellprogramme.org or email beewell@manchester.ac.uk.  

91ֱ�� researchers, based at Saint Mary’s Hospital, part of Manchester University NHS Foundation Trust (MFT), worked with The University of Manchester and 91ֱ��-based firm genedrive Plc on the Pharmacogenetics to Avoid Loss of Hearing (PALOH) study. Together, they developed the pioneering, rapid bedside genetic test which was .

Using a cheek swab, the test can identify in 26 minutes whether a critically ill baby admitted to intensive care has a gene change that could result in permanent hearing loss if they are treated with a common emergency antibiotic, Gentamicin.

While Gentamicin is used to safely treat approximately 100,000 babies a year, one in 500 babies carry the gene change that can lead to permanent hearing loss when given the antibiotic.

The new test means that babies found to have the genetic variant can be given an alternative antibiotic within the ‘golden hour’ and could save the hearing of 200 babies in England every year.

PALOH study lead, Professor Bill Newman, Consultant in Genomic Medicine at MFT and Professor of Translational Genomic Medicine at The University of Manchester, said: “I am delighted for the team to receive this recognition of their fantastic efforts and their innovative approach in bringing this test to fruition.”

The new swab test technique, which was piloted at MFT, replaces a test that traditionally took several days and is the first use of a rapid point of care genetic test in acute neonatal care.

Dr Ajit Mahaveer, Consultant Neonatologist, Rachel James, Senior Research Coordinator and Nicola Booth, Research Nurse Manager on the Newborn Intensive Care Unit at Saint Mary’s Hospital, attended the awards ceremony in London and accepted the award on behalf of the team.

Dr Mahaveer said: "I am incredibly proud to be part of the team who made this study a reality and to be recognised at this year’s New Statesman Positive Impact Awards. It’s an honour to accept the award on behalf of the team, knowing the work we have put into delivering this research will truly make a difference to hundreds of babies’ lives each year.

“As a doctor dealing daily with infection, my main concern was how easy and quickly the test was to conduct, as it’s important that we do not delay antibiotic treatment. Our experience of using this test has been very positive. It’s straight-forward, non-invasive and will have a huge impact on our patients’ lives.”

Professor Newman, Theme Co-Lead Lead for Rare Conditions, National Institute for Health and Care Research, 91ֱ�� Biomedical Research Centre, continued: “I am absolutely thrilled with the success of the study and that this test is now being used in routine clinical practice. This test will make a real difference, helping to ensure babies are not going to lose their hearing for a preventable reason.”

It is expected the test could save the NHS £5 million every year by reducing the need for other interventions, such as cochlear implants.

Professor Dame Sue Hill, Chief Scientific Officer for England and Senior Responsible Officer for Genomics in the NHS said: “This ground-breaking bedside test for detecting whether an antibiotic could cause deafness in babies in intensive care is another example of how the NHS is harnessing the power of genomic medicine to transform patient care. This award is a tribute to the hard work of Professor Bill Newman and his team in 91ֱ��.”

Dr Gino Miele, R&D Director, genedrive plc, said: “The collaboration of our company with the research and clinical team at MFT is a shining example of the NHS working with a commercial company to deliver real improvements in patient outcomes in a cost-effective way.”

Caption: Rachel James, Senior Research Coordinator; Nicola Booth, Research Nurse Manager, Newborn Intensive Care Unit and Dr Ajit Mahaveer, Consultant Neonatologist at Saint Mary’s Hospital, part of MFT receiving the award on behalf of the PALOH team. (Photo credit: New Statesman Positive Impact Awards)

Now in its seventh year, the campaign has seen exponential growth with over 275,000 primary and secondary school pupils signed up to participate this year. Thousands of schools and STEM organisations across the UK and internationally, will be sharing science on 14 June 2022. The University of Manchester will welcome primary and secondary pupils to their newly-opened Engineering Building for this celebratory event where pupils will demonstrate and discuss their scientific questions and evidence with hundreds of guests.

This year’s theme is Climate Action - a pertinent theme that captures the interest and curiosity of us all. The pupils have spent weeks gathering data, analysing, and drawing conclusions about a wide range of questions, including:

·       What is the best green energy source to power our school?

·       Does location affect the amount of air pollution?

·       How well do natural insulators protect against colder climates?

The event will be attended by the Lord Mayor of Manchester and Councillor Donna Ludford, alongside local business and education professionals. The guests will be encouraged to listen and question the pupils about their findings as part of this inclusive and non-competitive event.  

“We are honoured to have the support of so many STEM organisations, industry and educational partners. Without their support the campaign would not have continued to reach so many children, especially those in areas of high socioeconomic deprivation. I thank each one for their ongoing support and encourage each to consider how to strengthen their partnership with GSSfS in the future.” said Dr Lynne Bianchi, Campaign Director

In commemoration of their involvement in this year’s Great Science Share for Schools, The University of Manchester have partnered with City of Trees to gift every school attending their own tree.  The opportunity to plant a tree in their school grounds or gift their tree to another Greater 91ֱ�� School will be a lasting legacy of their involvement in the Great Science Share for Schools campaign.

“We are confident the children’s experience, in sharing their own scientific questions and investigations, alongside the gift of a tree, will encourage them to continue thinking about science and climate action in years to come. Their participation will set them on course to be future scientists and engineers contributing to solutions that mitigate the impacts of the climate emergency or contribute to a more sustainable way of living.” said Steph Hepworth, Campaign Manager, Great Science Share for Schools

The event will also host ‘Sybil the Whale’, an enormous lantern puppet created for the Littleborough Arts Festival Lantern Parade. Sybil is a life-size blue whale calf created to share narratives around Climate Change and encourage us all to imagine the consequences of sea level rise across the globe.

The GSSfS is unique in its approach to raising the profile of science enquiry in a wide range of schools and educational settings. Teachers explain how the campaign that stimulates more time for science in school, enables pupils to consider issues around Climate Change whilst taking the positive step to improve the sustainability of their school environment, through initiatives like this year’s tree planting.

Devised by researchers at The Universities of Manchester,  the system - costing under £2 per family for a week- was also shown to improve the overall quality of bedtime routines as well as parental mood.

The 50 first-time parents with children aged 1-3 were recruited to study received a variety of text messages for seven consecutive nights providing information on achieving optimal bedtime routines. The messages were co-designed with the parents.

A focus group of 25 of the parents was also held after the study was completed to gather feedback on the intervention.

They provided expressed their support and desire to see an intervention like the one trialled more widely available.

The results, obtained from pre and post intervention questionnaires, are published in the journal .

Funded by the Medical Research Council, the study team examined 6 key areas for achieving good bedtime routines: brushing teeth before bed; time consistency for going to bed; book reading before bed; avoiding food/drinks before bed; avoiding use of electronic devices before bed; and calming activities with child before bed including bath, shower, and talking.

As a result of the study:

• ·The children’s sleep increased by an average of 8% with less night-waking episodes and with children feeling better the next day after having a good night’s sleep
• ·The overall quality of bedtime routines improved by an average score of 4.8% with parents achieving more optimal, bedtime routine activities such as brushing teeth before bed, minimising consumption of sugar before bed, reading a book or storytelling before bed, avoiding use of electronic devices and interacting with their children more in calming, beneficial activities
• ·Parental mood was improved by an average score of 5.8% with parents reported feeling less tense, less fatigued and reporting higher self-esteem

Text messages have been used extensively within health behaviour change programmes, though they have never been used to deliver a standalone intervention for bedtime.

Dr Georgios Kitsaras who led the study said: “We know that there is strong link between the quality of bedtime routines and children’s sleep.

“We also know that poor sleep hygiene affects children’s development, school performance, mood and cognitive functioning and development as well as the wellbeing of parents.

“Organisations like the and are all engaged in this debate- but up to now, there has been sparse evidence of how best to help parents achieve better bedtime routines.

“Parents are on the receiving end of, at times, conflicting information and so we need to untie conflicting signals and messages parents receive.

“This lack of a clear consensus-based definition of limits health professionals’ ability to communicate best practice effectively with families.

“So any intervention which is shown to be effective is most welcome and could make a real difference to families.

“The low cost of the intervention, its adaptability and practicability also make it important in times of strained healthcare budgets and healthcare staff under pressure.”

He added: “The preliminary data from this low cost intervention is very encouraging:  we saw beneficial effects across three key outcomes: children’s sleep quality, bedtime routine quality and parental mood disturbance.

“And parents felt less tense, less fatigued, less confused, less angry while also reporting higher vigour and self-esteem.

“The impact on tooth brushing was particularly encouraging as  poor oral hygiene practices can increase the likelihood of tooth and gum disease.

“In England alone, 30% 3 year-olds and 25% of 5-year-olds have active dental disease, leading, at times, to extraction under general anesthetic

“It is therefore essential to ensure that all children have good oral hygiene practices and limit their exposure to sugar at all times, including at bedtime.”

Bedtime Routines Intervention for Children (BRIC) project: results from a non-randomized feasibility, proof-of concept study is published in

Taking just 25 minutes, the bedside machine identifies whether a critically ill baby admitted to intensive care has a gene that could result in permanent hearing loss if they are treated with a common emergency antibiotic.

The new swab test technique would replace a test that traditionally took several days and could save the hearing of 180 babies in England alone every year.

People admitted to intensive care are usually given an antibiotic called Gentamicin within 60 minutes. While Gentamicin is used to safely treat about 100,000 babies a year, one in 500 babies carry the gene that can make it cause permanent hearing loss.

Developed in 91ֱ��, the new test means that babies found to have the genetic variant can be given an alternative antibiotic within the ‘golden hour.’

It is expected the test could save the NHS £5 million every year by reducing the need for other interventions, such as cochlear implants.

First-year nursing student Mary, from Preston, is mother to 18-month-old Khobi, who was born and treated at Saint Mary’s Hospital, part of Manchester University NHS Foundation Trust.

Mary said: “Khobi was born with her bowel outside her tummy, which put her at risk of infection - she needed antibiotics quickly but was given this new genetic test which showed she was susceptible to hearing loss from gentamicin.

“She was given an alternative antibiotic which didn’t affect her hearing, and it worked well. She’s doing fine and is such a happy, sociable baby.

“This test is great, and I think all babies should have it.”

NHS national medical director Stephen Powis said: “The successful trial of this bedside test is fantastic news for the hundreds of babies - and their parents - who would otherwise lose their hearing when given this common antibiotic in intensive care situations.

“Through world-class innovation, the NHS is delivering cutting edge treatments to save and improve patients’ lives as well as delivering on the commitments of the NHS Long Term Plan.”

Professor Bill Newman, a consultant in genomic medicine at 91ֱ�� University NHS Foundation Trust and Professor of Translational Genomic Medicine at the University of Manchester, led the Pharmacogenetics to Avoid Loss of Hearing (PALoH) study. He said: “I am absolutely thrilled with the success of the study, and that this testing is now going to be used in three of our Trust’s Neonatal Intensive Care Units -  it’s actually going to make a real difference so babies are not going to lose their hearing for a preventable reason.

“The trial demonstrated that you can deploy rapid genetic testing in a clinical setting, and that the tests can be carried out within the ‘golden hour’ when severely unwell babies should be treated with antibiotics.”

Following the completion of the ground-breaking study, the NHS Genomic Medicine Service Alliance and the NHS will be exploring how this technology can be launched as part of a clinical service through the NHS Genomic Medicine Service.

Around 300 nurses are being trained to use the machine across MFT at Saint Mary’s Hospital, Wythenshawe Hospital, and North 91ֱ�� General Hospital, and the test is expected to be routinely used in all the hospitals’ neonatal units within weeks, which are part of Saint Mary’s Managed Clinical Service within the Trust.

Professor Dame Sue Hill, Chief Scientific Officer for England and Senior Responsible Officer for Genomics in the NHS, said: “Genomic medicine is transforming healthcare, and this is a powerful example of how genetic testing can now be done extremely quickly and become a vital part of triage - not only in intensive care but across our services.

“It also shows the importance of thinking about how advances in technology can rapidly transform how we use genomics closer to care for our patients.”

Professor Newman, who is Associate Lead, Hearing Health Genomic Solutions at NIHR 91ֱ�� Biomedical Research Centre (BRC), said the team had successfully transferred the accuracy of the machine in the lab to working effectively in a ward. At the same time, clinicians adapted quickly to incorporate the test into their routine care for very sick children in the neonatal ward.

The idea for the test came five years ago, and trials started in 2020 with further adaptations and fine-tuning so that the current machine, which is called the Genedrive System, is now fully CE certified to be used in a clinical setting.

91ֱ�� – the top-ranked paediatrics journal in the world – last week.

Backed by £900,000 funding from the National Institute for Health Research (NIHR) and support from the charity Royal National Institute for Deaf People, the Genedrive System, which costs £80 per baby, was developed by Genedrive, a start-up based at the University of Manchester. It was developed in close collaboration with Professor Newman’s team at 91ֱ�� Biomedical Research Centre.

Mike Hobday, Director of Policy and Campaigns at the National Deaf Children’s Society, said: “The National Deaf Children’s Society welcomes the publication of this important study. The introduction of a rapid test to identify susceptibility to deafness caused by the antibiotic gentamicin will be greatly valued by many families of newborn babies.

“Up to now the genetic test has taken too long to return from the lab to be useful for babies requiring urgent treatment but a rapid test will be a game-changer.”

David Budd, CEO of Genedrive, said: “There is a significant drive within the NHS to alert healthcare professionals to the impact of antibiotic-induced hearing loss and encourage them to consider genetic testing prior to initiation of treatment. 

“It’s a great example of using human genetics to guide specific therapy, which is now taking front and centre in clinical management globally. The application of Genedrive’s technology shows how a rapid, affordable, point-of-care test could impact patients’ treatment and quality of life across this as well as a wide range of fields.”

The study from 25 countries led by University of Manchester and Boston Children’s Hospital scientists, is published in Annals of the Rheumatic Diseases

Of the 607 patients, 378 had juvenile idiopathic arthritis (JIA), 78 had auto-inflammatory syndromes, and 47 had systemic lupus erythematosus or mixed connective tissue disease.

Most patients did not report any comorbidities (83%), though 38 (6%) had eye inflammation, a common condition in children with juvenile-onset arthritis.

The study also found that those on anti-rheumatic “biologic” therapies, such as TNF inhibitors, did not appear to be at meaningfully increased risk of developing severe COVID-19, compared to other children in the study who were not receiving these drugs.

The data used by the team was entered by doctors into the European Alliance of Associations for Rheumatology (EULAR) COVID-19 Registry, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry, and the CARRA-sponsored COVID-19 Global Pediatric Rheumatology Database.

All cases of COVID-19 occurred before vaccinations were available in the young people in this study.

Dr Lianne Kearsley-Fleet, an epidemiologist at The University of Manchester, said: “Previous research has shown that most children and young people do not experience severe COVID-19, many being asymptomatic or with only mild symptoms.

“So we felt it was important to find out if the same was true for those with RMDs, and the good news is that most do appear to do well and experience mild COVID-19 disease.”

Min-Lee Chang, co-author of the paper who led the data analysis for the CARRA dataset from Boston Children’s Hospital, said: “We of course agree that protective measures are important to follow to minimise the risk of acquiring SARS-CoV-2 infection.

“However these findings should help reassure parents and families that the probability of severe COVID-19 in the majority of children and young people with JIA appears relatively low.

Though the majority of children did well, 43 patients (7%) were hospitalised.

Where hospitalisations did occur, they were more likely among those with more severe RMDs such as lupus, vasculitis, or auto-inflammatory syndromes, rather than JIA. As in other studies, those who were obese were four times more likely to be hospitalised.

However, even among those hospitalised, most patients avoided severe illness, with less than one-in-five needing oxygen or mechanical breathing support.

Professor Kimme Hyrich from The University of Manchester and a consultant rheumatologist, said: “The data are very reassuring but do show again the important association between obesity and more severe COVID-19 outcomes,  supporting the view that protection measures in those children should be strictly followed.”

Dr Marc Natter, assistant professor of paediatrics at Harvard Medical School and the paediatric rheumatologist leading the study for CARRA at Boston Children’s Hospital, said: “The collective experience is that children, especially younger children with juvenile idiopathic arthritis appear less susceptible to symptomatic severe COVID-19 than adults with rheumatic disease, and reports of severe disease and death .

“But until now, little was known about the impact of comorbidity and immunosuppression on the risk of severe COVID-19 in the paediatric population with RMDs.

“This paper offers an important addition to the literature and should be reassuring for young people living with RMDs and their parents, although it does also reinforce the need for understanding there appears to be an overall increased risk that does need to be managed by COVID-19 vaccination, social distancing, and masking where appropriate.”

The paper Outcomes of SARS-CoV-2 Infection among Children and Young People with Pre-existing Rheumatic and Musculoskeletal Diseases is available  

The qualitative study of eight  young people, eight  family members and eight nursing staff by psychologists from The University of Manchester and Pennine Care NHS Foundation Trust is published in high profile journal PLOS ONE.

The professional connection between a clinician  and a patient -  known as a therapeutic relationship - can help improve outcomes for mental health patients say the research team.

Progress in psychotherapy and mental health care in general has previously been shown to strongly link to the therapeutic relationship between clinical professionals and service users.

However, the study highlights how nursing staff sometimes do not have the time or support to develop therapeutic relationships with their patients.

To achieve that, the researchers urge the employment of adequate staff numbers, focused training and time in cultivating connections between nursing staff and their patients.

“This research underlines the established point that therapeutic relationships between patients and staff are just as important as the specific treatment they are receiving, if not more so,” said Dr Sam Hartley, an honorary clinical lecturer  at The University of Manchester and Principal Clinical Psychologist with Pennine Care NHS Foundation Trust.

The young people, all based within child and adolescent mental health services across four sites in the UK, described how their relationships with nursing staff could impact on their progress through treatment.

The researchers interviewed the participants at length, and identified six themes which described therapeutic relationships, their development and maintenance.

One of the themes was centred around the feeling that therapeutic relationships are a treatment in their own right.

Dr Harley said: “Therapeutic relationships  are particularly pertinent in child and adolescent mental health inpatient services where relationships are especially complex and difficult to develop and maintain.

“Our analysis indicates that young people, families and nursing staff all agree these relationships are crucial to good outcomes. These groups would be better served by a system that prioritises the formation and maintenance of effective therapeutic relationships.

“This requires adequate staff numbers, training and time in cultivating connection and doing ‘normal’ things together.

“Consideration should also be given to aspects of the workforce that might impact on this being successful, such as staff retention, where continuity of care and relationships might be impeded.”

She added: “The balance between being human and professional is a tricky one and could benefit from ‘live’ focused staff support alongside more static training and supervision.

“We hope that the testimonies of these patients, nurses and parents, and our analysis will serve to drive policy makers, service managers and clinicians to focus on therapeutic relationships, as essential to quality inpatient care, and afford them the structures, support and significance they deserve.”

Citation: Hartley S, Redmond T, Berry K (2022) Therapeutic relationships within child and adolescent mental health inpatient services: A qualitative exploration of the experiences of young people, family members and nursing staff. PLOS ONE 17(1): e0262070. 

An animated video summary is available

The research, published in the , was funded by organisations including, the ,  and .

The international collaboration was led by Professor Bill Newman, Consultant at 91ֱ�� University NHS Foundation Trust, and Genomic Solutions Associate Lead for  theme.

The theme improves the lives of adults and children by preventing potentially devastating congenital deafness, diagnosing acquired age-related hearing deficits, and developing new treatments. Professor Ray O'Keefe, Professor of Molecular Genetics at the University of Manchester co-led the study.

Professor Newman, who is also Professor of Translational Genomic Medicine in  at The University of Manchester said: “Providing more families with an accurate diagnosis for their child’s health problems removes the need for unnecessary investigations, allows closer monitoring to spot problems earlier – and enables accurate genetic counselling for other family members who may be at risk.

“This research began 10 years ago at , when we saw a local family who had Perrault syndrome. We undertook some genetic studies and identified a novel gene and changes in that gene that caused that diagnosis in that family.

“For a lot of genetic conditions there is just one gene that is responsible for those health problems. After sharing our discovery we had requests from around the world asking us to undertake tests in their patients, to see if they had changes in this same gene.

“Although patients had the same conditions as the 91ֱ�� family, we weren't finding changes in this same gene. That made us think there must be other genes involved. Working with others around the world we have now identified eight different genes that can cause this same condition.

“We know these genes are important in a part of the cell called the mitochondria, known as the energy bundles of the cell, we know that some tissues in our body are very susceptible to when the mitochondria don't work, and that's why we believe these hearing and ovarian problems occur.

“As girls do not usually receive this diagnosis until puberty, earlier diagnosis would help young women to make decisions about preserving their eggs before menopause, to allow reproduction options later in life. 

“Babies with significant hearing loss will now be screened for changes in these genes so that we can identify earlier if they have Perrault syndrome. This has potential life-changing impact for families.”

Dr Ralph Holme, Director of Research and Insight at RNID said: “We are delighted to have been able to help fund this important research. Not only will it directly benefit families with this specific type of hearing loss, but a deeper understanding of the biological processes involved in hearing are likely to have wider implications, providing insights into more common forms of hearing loss.

Professor Newman added: “Understanding that many different genes and changes in them can cause the same condition helps us to think in terms of new specific treatments.

“Going forward, knowing that these genes are all linked together means that perhaps it would be possible to create a treatment that would work for all of them.”

The study of parents and children coping with conditions that cause adrenal insufficiency – such as and – - showed that administration of potentially lifesaving injections given at the time of acute illness is poor.

Researchers studied the medical data of around 300 young people under the age of 25 with adrenal insufficiency, who were cared for at MFT’s Royal 91ֱ�� Children’s Hospital (RMCH) and Royal 91ֱ�� Infirmary (MRI) between 2006 and 2019.

Their retrospective analysis revealed that during this period, 13 young people, with a median age of 10, died from causes likely related to adrenal insufficiency a rate of one death per year. The findings were .

Though little scientific evidence exists on the prevalence of adrenal death, the figure is higher than the number of excess deaths in children with Type 1 Diabetes, and much higher than for children with no chronic health conditions.

People with adrenal insufficiency need to take daily steroid medication, such as hydrocortisone, to replace the hormone cortisol, which is normally produced by the adrenal glands and helps to control the body’s blood sugar levels and other vital functions.

Parents and carers are given extensive advice about increasing hydrocortisone doses when children are unwell to avoid adrenal crisis, a task successfully carried out by parents who took part in the study.

Parents are also trained to give emergency steroid injections when children are seriously unwell but the need for this for each child is rare and may happen many years after training.

“Sometimes adrenal crises can happen very quickly and parents understandably call 999 or rush to A&E rather than administer the emergency injection,” said Dr Chris Worth, Doctoral Researcher at The University of Manchester and Clinical Research Fellow at Royal 91ֱ�� Children’s Hospital.

“But our study revealed that only  two of 17 patients received an emergency injection of hydrocortisone at home before attendance in A&E.”

The study also revealed a mismatch between reported confidence in administering emergency hydrocortisone injections and the actual administration.

 Almost 79 per cent of parents said they were confident administering intramuscular hydrocortisone but only 20% identified a missed opportunity for injection.

“In children experiencing adrenal crisis, parents followed the guidance given to them in clinic for oral hydrocortisone, but rarely administered intramuscular hydrocortisone,” said Dr Worth.

“This finding is at odds with the 79 per cent of parents who reported confidence in giving the injections.”

He added: “We think local training programmes for management of adrenal crisis are comprehensive, but insufficient to prevent the most serious of cases.

“It is clear that existing methodology needs to be rethought, and that novel and more immersive learning and practical tools such as simulation models and virtual reality software may be necessary.

Peter Clayton, Professor of Child Health and Paediatrics at The University of Manchester and MFT Honorary Consultant in Paediatrics and Paediatric Endocrinology, said: “Adrenal insufficiency can lead to life-threatening adrenal crisis and adrenal death.

“Parents are trained to prevent, recognise and react to adrenal crisis but until now there has been little information on what parents are actually doing at home to manage this problem.

“These children often live ordinary lives and most of the time manage their condition well; it’s a terrible tragedy when a death occurs merely because they did not receive the steroid injection they so desperately needed.

“We need to rethink our approach to training – and ask how we can help parents to recognise when to give an injection and also how we help them remember to do this in a time of very high stress.”

The GOSH Charity and Sparks National Call is part of an ambition to help unlock breakthroughs in child medicine by supporting researchers’ investigations into the causes of rare diseases in children, and conditions that start in childhood. The funding will also help supercharge their efforts to discover new and better ways to diagnose, treat, and ultimately cure these life-changing and life-limiting conditions.

Researchers based at six institutions across the country will benefit from the cash boost, including The University of Manchester, University of Warwick, and University College London Great Ormond Street Institute of Child Health, University of Southampton, University of Oxford, and University of Cambridge.T

The successful projects to be chosen for funding include:

• Developing more effective and kinder treatment for Diamond-Blackfan Anaemia, a rare blood disorder that affects the bone marrow’s ability to produce red blood cells, and can cause problems with a child’s growth, immune system, and heart
• Identifying a new treatment to address the underlying mechanisms which cause Dravet Syndrome, a life-limiting form of epilepsy
• Uncovering the underlying mechanisms in acrodysostosis and developing a technique for testing new potential therapies
• Helping to better understand the pain experienced by children with childhood cancer, which could help manage their pain and enhance their quality of life

GOSH Charity and Sparks invited researchers from across the UK to apply for funding as part of its National Call. Of the £2.5 million pledged to support research into some of the most difficult and hard to treat childhood diseases, Sparks contributed £900,000.

£112,500 has been made available by two condition-specific partner charities (Acrodysostosis Support & Research, and Dravet Syndrome UK) to help co-fund research into these diseases.

 Louise Parkes, Chief Executive at GOSH Charity, said: “The impact of research has never been more visible than over the past year, following the development of the COVID-19 vaccine. It shows that essential funding into research can have a life-changing effect on so many people. We’re thrilled that this year’s GOSH Charity and Sparks National Call is investing over £2.5 million into paediatric research projects, with huge thanks to our partner charities, without whom we wouldn’t be able to deliver the National Call. These projects have the potential to deliver kinder and more effective treatments for some of the rarest and most complex conditions and, more importantly, offer children and their families hope for a better future.”

Dr Siddarth Banka from The University of Manchester who has received funding said: “I am delighted to have received funding from GOSH Charity which will enable us to further our research to help children with chromatin disorders like Kabuki syndrome and Kleefstra syndrome. It’s fantastic to know that the charity is making such a large amount available for child health researchers across the UK to bid for each year.”

The National Call reflects GOSH Charity’s commitment to paediatric research funding. To find out more about what research GOSH Charity funds, and how Great Ormond Street Hospital has been behind some of the biggest breakthroughs in child medicine, visit .

To find out more about the National Call, and to apply for next year’s funding please visit

The definition, agreed by 59 UK experts is published in - one of the world’s leading scientific journals – and will provide welcome guidance to parents who want the best for their children at bedtime.

Funded by the Medical Research Council, the definition identifies 6 key areas,:

• Brushing teeth before bed.
• Time consistency for going to bed.
• Book reading before bed.
• Avoiding food/drinks before bed.
• Avoiding use of electronic devices before bed.
• Calming activities with child before bed including bath, shower, and talking.

The study also devises two different ways of scoring bedtime routines: one which measures a single routine and the other for activities over 7 days

A parent should aim to score at over 50 points to achieve an effective routine, says Dr George Kitsaras who led the study.

The same scoring system is used for another ‘dynamic measurement’ where depending how many nights a week parents achieve the activities they receive different, weighted scores multiplied by 1.0.

Dr Kitsaras said: “Bedtime routines are important family activities and have important implications on children’s wellbeing, development and health.

“Organisations as diverse as the to the and the are all engaged in this debate- but up to now, there has been no real scientific consensus to inform them; we need untie the conflicting signals and messages parents receive.

“This lack of a clear consensus-based definition of limits health professionals’ ability to communicate best practice effectively with families.

“Our definition considers the parental stresses and difficulties that might arise at bedtime while incorporating best practice and available scientific advice.

“This study for the first time provides that expert and scientific guidance.”

The psychologists, dentists, public health specialists and other experts from education, health visiting and sleep research participated in what is known as a Delphi Process, a method of achieving wider consensus by collecting opinions through several rounds of questions.

Eleven experts took part in an initial group, followed by 25 in round 2, 20 in round 3 and 13 in round 4.

Dr Kitsaras added: “All activities around bedtime matter for children’s development and wellbeing. From the wide range of activities around bedtime, our experts considered toothbrushing to be the most important to remember each night.

“There are strong links between inadequate oral hygiene practices and dental decay in children and adults. For children, early childhood caries can lead to higher occurrence of dental disease in later life and, in some cases, untreated childhood caries can lead to extractions under general anaesthetic causing additional problems for children and parents.

“Washing or having a shower each night before bed, on the other hand might be a common practice for families but our experts considered it to be part of a wider umbrella of child-parent interactions rather than a standalone practice we need to specifically target.

“I have no doubt the debate will continue and our definition might even be refined as more people engage with it.”

The paper ‘Defining and measuring bedtime routines in families with young children; a DELPHI process for reaching wider consensus’ is published in

Photo by on

Early results for a potentially revolutionary brain disease therapy given under compassionate use to a 2 year-old has shown promise, a University of Manchester scientist will tell an international conference on Wednesday (13 May).

Professor Brian Bigger will tell the American Society of Gene & Cell Therapy that the safety and feasibility of the investigational gene therapy for the rare and life limiting Sanfilippo disease type A is now becoming more clear, as a formal clinical trial gets underway.

The trial is funded by Orchard Therapeutics, who hold the license and commercial rights to the Sanfilippo disease type A programme.

The investigational gene therapy, which involves harvesting patient’s blood stem cells, was delivered by Professor Rob Wynn under a specials licence at The Royal 91ֱ�� Children’s Hospital (RMCH) in 2019.

The therapy and trial follow over a decade of development and pre-clinical work by Prof Bigger at The University, together with clinical collaborators Prof Wynn, Prof Ed Wraith and Dr Simon Jones at 91ֱ�� University NHS Foundation Trust (MFT) and Prof Adrian Thrasher at Great Ormond Street Hospital (GOSH).

A type of virus known as a lentiviral vector is used to deliver the patient’s missing ‘SGSH’ gene to the blood stem cells in the laboratory.

The manufactured gene-modified stem cell product is transplanted back into the patient.

The stem cells then regenerate the immune system providing the patient with the working - and potentially permanent - copy of the gene.

Sanfilippo disease type A – or MPSIIIA - causes a progressive loss of developmental skills in young children.

They suffer deafness, hyperactivity and behavioural problems, progressive developmental delay, and seizures during the later stages of the condition.

The condition is usually fatal in late childhood or early adulthood.

Professor Bigger said: “These results are promising and provide some hope for these children whose condition was previously thought to be incurable. The trial is the critical next stage, but it’s true to say we are excited.

“Sanfilippo Disease is an appalling disease which causes misery to these children- so the prospect of a treatment is tantalising – especially when it was thought for years that no treatment would be possible.”

The preliminary results of the patient treated under compassionate use and followed now for over a year post-treatment has shown rapid engraftment of the gene modified cells and greater than 25-fold of median normal SGSH enzyme levels achieved in the blood. At 6 months post-treatment, CSF levels of SGSH enzyme were at the upper end of normal range.

Sufficient replacement of the enzyme by blood cells in regions such as the brain, could result in prevention of the disease course, but it is still too early to know if this has happened in this case.

Sanfilippo disease, is caused by defects in the , which is needed to break down complex sugars in the body. A lack of this enzyme leads to a build-up of these sugars in the body and this mainly affects normal brain function in these children.

In the phase I/II trial, the gene therapy transplant will be given to between 3 and 5 children, aged under 2 years with a severe MPSIIIA type.

The first patient was recruited to this trial in early January 2020, with their blood stem cells harvested and modified prior to transplanting the gene therapy product back into the patient in May 2020.

The safety of this investigational gene therapy, the level of SGSH enzyme and changes in patient behaviour and quality of life, alongside other measures, will be continually assessed over the next 3 years.

The medical team include Paediatric Haematologist Professor Robert Wynn and Dr Simon Jones from RMCH, who have experience of bone marrow transplantation for children with metabolic enzyme deficiencies.

Professor Wynn said: “RMCH has a long-standing expertise in cell therapy of metabolic diseases, and the original ideas and early attempts to use gene therapies in these diseases were three decades ago.

“This advance builds on that clinical experience and those ideas. We hope and expect that this gene therapy approach will make our treatments safer, more effective and, course, most importantly will lead to better lives for our patients.”

The trial is being sponsored by The University of Manchester and the gene therapy product is being manufactured at University College London Great Ormond Street Hospital Institute of Child Health (ICH) using the expertise of Professor Thrasher’s specialist gene therapy laboratories.

Prof Bigger added “The original funders of this work including the Society for Mucopolysaccharide Diseases, Great Ormond Street Children’s Hospital Charity (GOSH Charity) and the Sanfilippo Children’s Research fund who were instrumental in making this trial a reality. We are all very grateful for their support throughout the process.”

The manufacturing work on the study was supported by NIHR Great Ormond Street Hospital Biomedical Research Centre.

A new study reveals Italian-speaking children pick up some language rules faster than English-speakers.

Learning a language is more than just knowing the words – it’s knowing the rules too. A new paper from researchers at has investigated the speed at which Italian-speaking infants are able to pick up rules around singular and plural forms of words. They found that infants had learnt this distinction by 12 months.

The study is published in the journal

In contrast, previous research on this subject has found that English-speaking infants begin to understand singular/plural distinction at around 20 months.

The reason for this might be that the rules for pluralising words in Italian make more sense than those in English. For instance, changing “the yellow giraffe” to “the yellow giraffes” in Italian requires changes in three spots (“la giraffa gialla” becomes “le giraffe gialle”). While this might seem more complex than English, consider goose/geese versus moose or sheep!

In addition, the greater number of changes required between singular and plural in Italian may help reinforce the rule, improving how quickly it is learned.

“We know that all languages have words and that word learning seems to happen in the same way across the world.” Said Dr Alissa Ferry, one of the researchers involved in the study.

She continued; “But all languages have different rules about how words are put together and when children start to figure out those rules does seem to vary depending on the language.”

To investigate the speed at which infants pick up the Italian rules, the researchers had children aged either 12-, 18- or 24-months play a game. They were shown two pictures on a computer screen with either one or two people in each picture and then heard the word for singular (for example, la bambina, the girl) or plural (le bambine, the girls).

How long the infants spent looking at the correct picture was used to gage their understanding of language rules.

The results showed that, by 12 months, the Italian infants could correctly distinguish between girl or girls, depending on which form they heard. This means they were able to pick up the rules around eight months earlier than their English-speaking counterparts.

“We know that all languages have different rules, and these findings show that those rules can shape when the infants start to figure them out,” Said Dr Ferry, adding; “Rules that are harder to find, like the English plural system, take a bit longer to figure out that rules that are easy to find, like the Italian plural system.”

“Twelve to 24-month-olds can understand the meaning of morphological regularities in their language” is published in Developmental Psychology

Children of women who reported domestic violence in pregnancy or during the first six years of the child’s life are almost 50% more likely to have a low IQ at age 8, research finds.

In the study by University of Manchester epidemiologists, 13% of children whose mothers did not experience domestic violence had an IQ of below 90 at 8 years of age.

If their mothers experienced physical violence from their partner either in pregnancy or during the first six years of the child’s life, the figure rises to 22.8%.

The team led by Dr Kathryn Abel from The University of Manchester show the chance of a low IQ rises to 34.6% if the mother was repeatedly exposed to domestic violence.

That means children with mothers who repeatedly suffer domestic violence during pregnancy and the first six years of their child’s life are almost three times more likely to have a low IQ at 8 years of age, find researchers.

Low IQ is defined as an IQ score less than 90, where a normal IQ is considered to be 100.

The study examined the link between domestic violence - also called Intimate partner violence (IPV) - and child intelligence at 8 year’s old, using 3,997 mother child pairs from The University of Bristol’s Avon Longitudinal 91ֱ�� of Parents and Children.

The study, funded by the Wellcome Trust and Medical Research Council, is published in .

ALSPAC follows children from pregnancy, and measures emotional and physical domestic violence – also known as intimate partner violence - from pregnancy until eight years of age.

The intelligence of the children was measured at eight years using the Weschler standardised IQ test.

Dr Abel said: “We already know that 1 in 4 women age 16 and over in England and Wales will experience domestic violence in their lifetime and that their children are at greater risk of physical, social and behavioural problems.

“We also know that intelligence in childhood is strongly linked with doing well in adulthood, though there has been little evidence about the risk of low IQ for these children.

“While we cannot conclude that IPV causes low IQ, these findings demonstrate domestic violence has a measurable link, by mid-childhood, independent of other risk factors for low IQ.”

17.6% of the mothers in the study reported emotional violence and 6.8% reported physical violence.

The findings are independent of other risk factors for low IQ such as alcohol and tobacco use in pregnancy, maternal depression, low maternal education and financial hardship around the child’s birth.

There is some disagreement on whether the IQ test is a complete measure of intelligence, as it only considers verbal and non-verbal intelligence

However, it is regarded as useful by many experts because a high IQ has been demonstrated in many countries and cultures to associate with a broad range of improved social and health outcomes.

Dr Hein Heuvelman, from The University of Bristol added: “Exposure to domestic violence is common for children in the UK and an important and often overlooked risk factor in their life chances.

“So knowing the extent to which these already vulnerable children are further affected is a powerful argument for more, better and earlier intervention.

“Current support for women experiencing domestic violence is inadequate in some areas and absent in others.

“Early intervention with these families protects children from harm, but it may also prioritise their future development.”

The study, funded by The Wellcome Trust and Medical Research Council, was carried out by a team of experts at The University of Manchester, University of Bristol, 91ֱ�� Metropolitan University and Kings College London.

The paper ‘Intelligence in offspring born to women exposed to intimate partner violence: a population-based cohort study’, published in , is available 

Based at the University of Bristol, Children of the 90s, also known as the Avon Longitudinal 91ֱ�� of Parents and Children (ALSPAC), is a long-term health research project that enrolled more than 14,0000 pregnant women in 1991 and 1992. It has been following the health and development of the parents, their children and now their grandchildren in detail ever since. It receives core funding from the Medical Research Council, the Wellcome Trust and the University of Bristol.

National helplines:

Women's Aid – for women experiencing IPV freephone national 24 hour helpline 0808 2000 247 

RESPECT helpline for perpetrators of IPV to stop using violence and abuse 0808 802 4040

For media enquiries contact:

The devastating effects of a rare condition affecting new-born babies will be far worse if diagnosis and treatment are delayed.

Up to half of all babies born with congenital hyperinsulinism (CHI) - a disease that causes extremely low blood sugar - suffer from life-long disability.

But with more prompt detection and individualised management, patient outcomes are much more favourable, report the researchers in a new review published in Diabetic Medicine.

CHI - also coined the clinical opposite of diabetes - is the most common cause of persistently low blood sugar (hypoglycaemia) in early childhood.

Although generally rare, affecting just 1 in 50,000 children in the UK, CHI can be as common as cystic fibrosis (1 in 2,500) in some countries such as Saudi Arabia.

The research team from 91ֱ�� reviewed current therapies and outcomes of children and young adults with hypoglycaemia brought about by CHI.

According to the scientists, a hypoglycaemic episode - when blood sugar (glucose) concentration dips below 3.0 mmol/L - is harmful for the survival of nerve cells.

Treatment typically aims to make sure blood glucose does not drop lower than 3.3 to 3.8 mmol/L.

“Symptoms of low blood glucose in babies are very distressing. Babies are floppy, don’t feed very well and have a pale or bluish skin colour. It is really important that hypoglycaemia is recognised by parents and clinicians, and that it is treated quickly to prevent brain injury,” explained Doctor Karen Cosgrove, from The University of Manchester, who was involved in the study.

Scientists now understand that there is a critical window after birth in which an adequate energy supply is required for the correct development of the brain.

And that explains why new-borns are more prone to suffering from the life-long effects of low blood glucose than adults.

“Our initial data suggest that with earlier referral, the frequency of cases with adverse neurological development is reducing” explains Professor Mark Dunne from The University of Manchester and lead author on the study.

In healthy people, insulin brings down blood glucose when its concentration rises.

When an infant with CHI experiences a hypo - a hypoglycaemic episode - it is a result of their bodies releasing too much insulin.

This is unlike diabetes, where a hypo often occurs due to a mismatch between the patient’s glucose concentration and their medication.

Any form of hypo can be dangerous as glucose fuels vital organs in the body, especially the brain.

Mild symptoms such as the infant appearing drowsy or struggling to keep warm can quickly escalate into life-threatening seizures or comas if left untreated.

Repeated episodes of hypoglycaemia without prompt treatment may permanently affect the learning, memory and behaviour of a child.

Dr Karen Cosgrove added: “Providing early and individualised medical treatment for these babies is very important if we are to safeguard their health and improve their quality of life in the long-term.”

This review paper is available

To find out more about the impact that CHI has on families around the UK, visit For more information on one of the specialised UK services that treats CHI patients, visit

Children monitored regularly for height and weight are less likely to be overweight according to research by University of Manchester and Oxford experts.

Publishing in the journal Preventive Medicine Reports today, the researchers say current practice may fail to spot a large number of children who are a normal weight on school entry but develop obesity in later in their lives.

Typically, a single measurement is taken when children start school, which may not be repeated until they are aged 11; only children who are obese from a very young age are easily identified for support.

The data allowed them to plot population-level and individual-level growth curves for girls and boys and find typical patterns.

The study reviewed the literature and pooled data from 54 studies and over 750,000 children worldwide.

According to Dr Heather Robinson who conducted the research while at The University of Manchester, the research shows that children should be measured regularly from a young age to help prevent obesity in later life.

According to the review, since 2000 late increasing children have made up 5-19 percent of children in the UK, USA and Australia.

She said: “Adult BMI starts to be predictable from child growth patterns from as early as age 2 years in some children so we should be measuring them from that age.

“However, as most measurement programmes are linked to schools as a way of accessing large groups of children, we can still achieve much by making the most of these observations between 4 and 11 years of age.

“Continuing closures of Sure Start centres which measure UK babies could mean the few early years measurements parents get could soon be lost.

“Each year a number of parents indicate that the current format of the National Child Measurement Programme, which informs parents of children's underweight and overweight at age 4-5 and 10-11, is unhelpful.

“Our work reiterates that not only are a minority of children identified as obese when they are following healthy growth pathways, but a group of children who become obese later have BMIs in the normal range at 4-5 years, so are missed.”

Previous studies have shown that many health problems are linked to how trajectories of weight and height in early childhood.

This includes including adult obesity, metabolic syndrome, non–alcoholic fatty liver disease and type 1 diabetes.

The research also shows that children adopt distinctive growth patterns before or within the 4-11 year age range.

Dr Matt Sperrin, from The University of Manchester added: “The trajectories we discovered can be classified into types which can be used to predict later life health outcomes.

“High risk growth trajectories predisposing adult obesity diverge from less harmful trajectories by approximately age 5.

“So we therefore argue that children and their parents should be targeted for healthy lifestyles considerably before this point.”

Dr Rinita Dam, from the University of Oxford, said: “We need to find effective ways of communicating issues related to children’s growth to their parents.

“We feel that by proactively encouraging and supporting parents and children to talk about this sensitive subject in a non-judgemental environment, much can be achieved.”

Dr Matt Sperrin, from The University of Manchesteradded: “The trajectories we discovered can be classified into types which can be used to predict later life health outcomes.

“High risk growth trajectories predisposing adult obesity diverge from less harmful trajectories by approximately age 5.

“So we therefore argue that children and their parents should be targeted for healthy lifestyles considerably before this point.”

Dr Rinita Dam, from the University of Oxford, said: “We need to find effective ways of communicating issues related to children’s growth to their parents.

“We feel that by proactively encouraging and supporting parents and children to talk about this sensitive subject in a non-judgemental environment, much can be achieved.”

A team at The University of Manchester have developed a novel stem cell gene therapy approach to treat children with a devastating genetic disease. The approach is currently being developed for clinical trial in patients with the disease.

Hunter disease affects the bones, joints, hearts and lungs of affected children and in about two thirds of cases also affects the brain, leading to severe mental disabilities. It is a genetic disease inherited by affected boys, caused by the missing IDS gene.

Although there is an existing enzyme replacement therapy available currently on the NHS, it costs more than £150,000 per year per patient and doesn’t penetrate to the brain.

’s team at The University of Manchester developed a stem cell gene therapy approach that works by replacing the missing gene in the bone marrow of affected children. To make this more effective, they also added a tag to the IDS enzyme to allow it to pass into the brain.

The team demonstrated complete correction of both the bone, joint and brain disease in mice, recently published in the journal .

Prof Bigger said “We expected the stem cell gene therapy approach to deliver IDS enzyme to the brain, as we have shown previously for another disease: Sanfilippo types A and B, but we were really surprised to discover how much better the tag made the therapy in the brain.

“It turns out that the tag didn’t only improve enzyme uptake across the blood brain barrier, but also improved uptake of the enzyme into cells and it appeared to be more stable in the bloodstream – all improvements on current technology.”

The team are part of the recently awarded InnovateUK 91ֱ�� Advanced Therapy Centre Hub (iMATCH), and aim to develop this therapy within this framework to take it to clinical trial.

A video describing the effects of the disease and potential of gene therapy can be seen  in Living Beyond Hope: A Short Documentary by Joshua Davies.

He added: “We are extremely grateful to our charity funders without whom this work would not have been possible, including the Isaac Foundation, the National MPS Society and the Newlife Foundation.

Ther image isn used on the cover of the journal .

University of Manchester scientists have developed a new gene therapy they hope will treat children with a rare but devastating brain disease, and plan to take it to clinical trial in the near future.

US-based biotech . have signed a licence deal with The University of Manchester, though its IP commercialisation company UMI3 Ltd, to take the treatment to the next stage, which will involve a clinical trial on patients with Sanfilippo disease type C.

Sanfilippo disease type C is a rare inherited neurodegenerative lysosomal storage disease caused by mutations in HGSNAT.

The technology is developed by ’s laboratory in collaboration with Dr. Els Henckaerts’ laboratory at King’s College London, and recently published in the journal Brain

It involves the use of a specially modified virus called adeno-associated viral vector (AAV), which has been specifically altered to efficiently deliver the missing HGSNAT gene to the brain to treat the disease.

Working with an international group of scientists, the team was able to demonstrate complete behavioural and brain correction of Sanfilippo disease type C in mice.

The King’s College London technology is based on the discovery of a novel AAV vector with an altered protein coat, which makes the virus work better within the brain. This new vector is called AAV-TT (AAV-true type).

The technology works better than the AAV9 vector, currently the gold standard for gene delivery to the brain.

Sanfilippo C disease affects children as early as 3 years of age, resulting in severe and rapidly progressive brain disease and neurological symptoms.

There is currently no effective treatment option for Sanfilippo disease type C as the protein is transmembrane and cannot move between cells.

This means that maximal vector distribution within the brain is critical for treatment success.

Prof Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester, said “This gene therapy technology recently published in the journal Brain, will be used by Phoenix Nest to treat Sanfilippo syndrome Type C.

“Sanfilippo is an incredible challenge as you have to be able to treat so many cells in the brain for complete success.

“In this work, the combination of the true type vector with improved brain distribution and the method of delivery were both critical for success.

“We were really impressed that we were able to completely correct working memory and hyperactivity in the mouse model – traits shared by children with the disease.

“Working together with Phoenix Nest Inc we hope this therapy will be successful in treating children with MPSIIIC in the next few years.”

“The study was funded by MRC, King’s College Commercialisation Institute, Jonah’s Just Begun, Sanfilippo Sud, Sanfilippo Barcelona, Sanfilipo Portugal, Sanfilippo Brasil, Le Combat de Haitem-Contre Sanfilippo, JLK- Sanfilippo Research Foundation, Sanfilippo Children’s Foundation, and VML charities and it has been great working with them towards a cure for this horrible disease.”

A University of Manchester historian is to highlight the untold experiences of children who were admitted to hospital in the early years of the NHS.

In a special lecture on April 24 supported by the NHS Confederation - marking the NHS's 70th anniversary of the launch of the National Health Service - will explore the human stories which define the NHS of the past and future.

Dr Snow is creating the first shared social history of the NHS, by collecting recordings, photos and other memorabilia, charting the momentous changes in the UK’s healthcare system since 1948, through ‘NHS at 70: The Story of Our Lives’ initiative.

She will tell how the NHS created new opportunities for fulltime paediatricians, culminating the 1962 Hospital Plan to include paediatrics as an essential service in all hospitals.

But she will also recount how parents were not allowed full visiting rights to their children, causing terrible distress to families.

It was only when Sir Harry Platt, Professor of Orthopaedic Surgery, at The University of Manchester and President of the Royal College of Surgeons reviewed arrangements for children in hospital in 1956 when things began to change.

Sir Harry recommended unlimited parental visiting though even by 1962, Platt said that ‘tradition and the difficulty of adapting existing hospital structures have curbed the pace of reform’.

Patricia Silverman was a 5-year-old child in hospital in 1948. She told Dr Snow: “I got upset in the hospital so they banned my mum and dad from seeing me. Can you believe that?

“I remember one day I saw them peeping round from behind a screen at the entrance to the ward I just went mad.

“They disappeared because they were scared and didn’t want to upset the staff

“I can remember running all over the hospital but someone must have caught me and taken me back.”

And Elizabeth Scanlon worked as a children’s nurse at Booth Hall Children’s Hospital in the 1950s. She later became involved with the National Association for the Welfare of Children in Hospital (NAWTCH) now known as Action for Sick Children.

She said: “The children became our responsibility when they came into hospital. I can remember the children being really upset when their parents were leaving.

“We had to go and comfort the children and say: ‘It’s alright, we’re going to look after you.’

“It was military almost, I suppose. That’s why I joined NAWCH whose main aim was to get parents access to children at all times.”

Dr Snow said: “NHS at 70: The Story of Our Lives is about capturing the seventieth anniversary of the NHS as a catalyst for a conscious focus on its past.

“And the NHS has an advantage over other significant historical anniversaries, because we still have access to people who participated in its 1948 creation

“But the timer is fast running out on the opportunity we have to collect this history, especially from the earliest years.”

She added: “Part of that narrative is the need to build children's experiences into the wider history of the NHS

“People like Patricia and Elizabeth’s testimonies will give us not just a richer understanding of children's experiences but also help modern medicine more responsive to children's needs.”

The NHS at 70: Voices from the past and visions of the future lecture will take place at University Place, University of Manchester, Oxford Road at 7.45pm. More details

NHS at 70: The Story of Our Lives project is supported by the Heritage Lottery Fund and based at the University of Manchester’s

A new test developed by University of Manchester and NHS scientists could revolutionise the way children with growth hormone deficiency are diagnosed.

Children suspected of having GHD – which cause growth to slow down or stop and other serious physical problems - currently require a test involving fasting for up to 12 hours.

The fasting is followed by an intravenous infusion in hospital and up to 10 blood tests over half a day to measure growth hormone production.

Because the current test is unreliable, it often has to be done twice before growth hormone injections can be prescribed.

Now the discovery - which the team think could be available within 2 to 5 years -could reduce the process to a single blood test, freeing up valuable time and space for the NHS.

from The University of Manchester and Dr Philip Murray from 91ֱ�� University NHS Foundation Trust, were part of the team whose results are published in the Journal of Clinical Investigation Insight today.

Dr Stevens said: “We think this is an important development in the way doctors will be able to diagnose growth hormone deficiency – a condition which causes distress to many thousands of children in the UK

“This sort of diagnostic would not be available even a few years ago but thanks to the enormous computing power we have, and advances in genetics, it is now possible for this aspect of care to be made so much easier for patients – and the NHS.

“These volume of data involved is so huge and complicated that traditional data-processing application software is inadequate to deal with it.”

Comparing data from 72 patients with GHD and 26 healthy children, they used high powered computers to examine 30,000 genes - the full gene expression- of each child.

A sophisticated mathematical technique called Random Forest Analysis analysed around three million separate data points to compare different gene patterns between the children with and without GHD.

The research identified 347 genes which when analysed with the computer algorithm can determine whether a child has GHD or not and thus whether they will benefit from treatment.

Growth hormone deficiency (GHD) occurs when the pituitary gland - which is size of a pea- fails to produce enough growth hormone. It more commonly affects children than adults.

Many teenagers with GHD have poor bone strength, fatigue and lack stamina as well as depression, lack of concentration, poor memory and anxiety problems.

GHD occurs in roughly 1 in 5,000 people. Since the mid-1980s, synthetic growth hormones have been successfully used to treat children - and adults - with the deficiency.

Dr Murray added: “This study provides strong proof of concept, but before it is in a position to be adopted by the NHS, we must carry out a further validation exercise which will involve comparing our new diagnostic with the existing test.

“Once we have crossed that hurdle, we hope to be in a position for this to be adopted within 2 to 5 years – and that can’t come soon enough for these children.”

Child Growth Foundation manager Jenny Child's daughter has Growth Hormone Deficiency.

She said: Growth Hormone Deficiency isn’t just about growth, as lack of growth hormone impacts the child in many ways, such as lack of strength and they can find it difficult to keep up physically with their peers. It impacts the child’s self-esteem as they are often treated as being much younger, because of their size. Growth hormone treatment allows the child to grow to their genetic potential.

"A growth hormone stimulation test can be very daunting for both child and parents.The test can make the child feel quite unwell and they can experience headaches, nausea and unconsciousness through hypoglycaemia."

‘’ is published in the Journal of Clinical Investigation - Insight.

Visit  for more information.