Our commitment to research into rare diseases

Great Ormond Street Hospital (GOSH) treats more than 28,000 children with rare diseases. With a third of children with a rare disease dying before their fifth birthday, investing in research is crucial.

Research forms a vital part of our 5-year strategy. We're committed to working alongside GOSH, our partners and our supporters to develop effective treatments and cures for children across the UK and beyond.

We know we can’t tackle rare diseases alone. With GOSH and our research partners, we can drive forward the development of effective treatments.

One of our key research partners is the independent medical research charity LifeArc.

LifeArc dedicates itself to transforming promising life science ideas into life-changing medical breakthroughs for patients.

Together we fund projects to accelerate progress of new discoveries so they can be developed into diagnostics, treatments and cures for rare and complex diseases. Our collaborative approach has helped fund seven studies over the last two years.

One of these studies is led by Professor Jane Sowden of UCL Great Ormond Street Institute of Child Health. Professor Sowden and her team are developing a cutting-edge stem cell therapy for children with inherited eye diseases. By replacing the damaged light-sensing cells in the retina at the back of the eye, this therapy has the potential of restoring sight and offering hope of a treatment.

For Rare Disease Day 2022, GOSH Charity’s Director of Impact and Charitable Programmes, Dr Kiki Syrad, wrote about the importance of collaboration in curing rare diseases . You can read her article here.

GOSH Charity is the UK’s largest dedicated charitable funder of child health, but medical research for children remains severely underfunded. If you’d like to support research and help make a difference to the lives of seriously ill children across the UK, please give what you can to our research fund.

Keep an eye on our social media channels this week to hear from some of our amazing GOSH families affected by rare diseases. You can also stay up to date with activity at GOSH by visiting the hospital website.