Dr Rajvinder Karda - exploration of a novel gene therapy to treat the devastating and life-limiting neurological condition Dravet Syndrome.
Great Ormond Street Hospital Children’s Charity (GOSH Charity) and Sparks, the children’s medical research charity have announced a £2.3million investment into 11 pioneering child health research projects. The funding is the UK’s largest charitable grant-making scheme of its kind, dedicated to paediatric rare disease research.

The cash boost will provide much-needed support to researchers’ investigations into the causes of rare diseases in children and will supercharge their efforts to discover new and better ways to diagnose, treat, and ultimately cure these life-changing and life-limiting conditions.

GOSH Charity and Sparks invited researchers to apply for funding as part of their ‘national call’. Of the £2.3 million pledged to support research into some of the most difficult and hard to treat childhood diseases, £182,926 has been made available by three condition-specific charities (the Norrie Disease Foundation, Dravet Syndrome UK, and the Myotubular Trust) to help co-fund research into these diseases.

Dr Hassan Rashidi – developing an implantable and removable liver ‘patch’ that could hold the key to reducing toxic chemical levels in the blood of children with rare metabolic conditions.

Based at six institutions including Cambridge University, the University of Oxford and the UCL Great Ormond Street Institute of Child Health, the researchers aim to improve diagnosis and develop more effective and kinder treatments for children who desperately need them.

Dr Giovanni Baranello – supporting the UK arm of a global clinical trial to test whether the breast cancer drug tamoxifen could help children with a rare and debilitating muscle disorder.

The successful projects include unravelling the drivers of aggressive childhood brain tumours; supporting the UK arm of a global clinical trial to test whether the breast cancer drug tamoxifen could help children with a rare and debilitating muscle disorder; exploration of a novel gene therapy to treat the devastating and life-limiting neurological condition Dravet Syndrome, and developing an implantable and removable liver ‘patch’ that could hold the key to reducing toxic chemical levels in the blood of children with a rare metabolic conditions.

Kiki Syrad, Director of Grants and Impact at GOSH Charity and Sparks charity, said:

“Year on year we are amazed by the quality and diversity of applications we receive from researchers across the UK and the life-changing potential their projects have. I am excited to see how this year’s projects progress.

“Collaboration in the field of rare disease research is of fundamental importance, so I am also delighted that GOSH Charity and Sparks have been able to partner with other rare disease charities through this funding call. We will achieve more together than we could alone and that is crucial if we’re going to drive new, urgently needed ways to diagnose, treat and cure rare diseases that affect children.”

The commitment to paediatric research funding reflects GOSH Charity and Sparks’ ambitions to help unlock breakthroughs in children’s medicines that will find tests, treatments and cures for seriously ill children with rare and complex conditions. Currently, paediatric research is severely underfunded, receiving only five per cent of public and charitable research funding in the UK each year.