Nine child health researchers funded by 2024/2025 National Funding Call
30 Apr 2025, 2:16 p.m.
As the UK’s largest dedicated charitable funder of child health research, we’re excited to announce that we’re funding nine new research projects.
The projects will help us to better understand and improve outcomes for rare or complex child health conditions.
£2.2 million has been committed through Great Ormond Street Hospital Charity’s (GOSH Charity) National Funding call - the UK’s largest annual funding scheme dedicated towards research into rare or complex childhood diseases.
The awarded projects come from organisations across the UK – including the University of Edinburgh, University of Birmingham and several based in London, including the UCL Great Ormond Street Institute of Child Health (UCL GOS ICH).
All projects were assessed by an expert scientific panel and the GOSH Charity Research Patient Insight Panel of Lived Experience Experts. These groups ensure that the highest quality projects with the maximum potential impact for patients and their families are funded.
The power of discovery research
Around half of the projects being funded involve discovery research. By learning what causes rare childhood diseases, researchers can find new ways to diagnose, treat and even prevent illness.
This knowledge is vital to future breakthroughs, like discovering new targets for better treatments and cures.
The projects will explore diseases such as childhood cancer, as well as other rare conditions that cause a wide range of symptoms, including joint abnormalities, progressive liver failure and learning difficulties.
"I am delighted to have been awarded a research grant from the GOSH Charity National Call. I am devoted to finding a cure for rare genetic disorders – an often-underappreciated health challenge. This grant will enable me to grow my recently established group at the Institute of Genetics and Cancer, University of Edinburgh. I aim to investigate therapeutic options for ATR-X syndrome, a rare genetic disorder associated with learning difficulties and other neurodevelopment delays.”
Bringing treatments one-step closer to patients
The remaining projects focus on cell and gene therapy. They aim to tackle conditions including cancer, liver disease, juvenile Parkinson’s disease, as well as other rare neurodevelopmental and metabolic conditions.
Cell and gene therapies can be life-changing for people who are often left with no other treatment options. Since around 80% of rare diseases are genetic, and almost 70% of those affect children, these therapies could revolutionise treatment of childhood rare diseases by targeting the cause of the condition, rather than simply managing the symptoms.
One project will test a gene therapy to prevent hearing loss in patients with Norrie Disease. This rare genetic condition that almost always affects boys causes blindness from birth and progressive hearing loss.
Currently, no treatments exist to prevent progressive hearing loss in Norrie disease, which can really affect a child’s independence and quality of life. This project is being funded in partnership with The Norrie Disease Foundation.
“Hearing loss has a huge impact on the mental health of the Norrie community, leaving patients feeling isolated and lonely. This research, that aims to save the vital hearing of an already blind community, could mean the difference of living independently or not as adults, and give patients a better chance of finding employment; feeling included in social settings; crossing roads more safely and enjoying music.”
2025/26 National Funding Call now open
Our ambition to transform the lives of seriously ill children through research-led care continues to grow. In the 2025/26 National Research Project Grant call (formerly known as the National Call) researchers can apply for up to £350,000 to study rare or complex childhood diseases.
There is £2.5 million available as part of our £70 million 2023-2028 research strategy. Through this strategy, GOSH Charity is committed to accelerating child health research – until no childhood is lost to serious illness.
Building on the success of previous rounds, we’ll continue to partner with other charities to jointly award funding for exceptional projects in specific disease areas. This year we’re proud to partner with Action for A-T, Cure DHDDS, Myotubular Trust, The Norrie Disease Foundation, Nystagmus Network and The Lily Foundation.
So, if you’re a child-health researcher looking for funding, find out more and apply to the 25/26 GOSH Charity National Research Project Grant call. The submission deadline is 3pm on Thursday 26 June 2025.