UK Charity Awards £2.5M to 11 Projects Into Rare Childhood Diseases

Vanda Pinto, PhD avatar

by Vanda Pinto, PhD |

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GOSH rare diseases children/ project funding


Great Ormond Street Hospital (GOSH) Charity and Sparks are investing £2.5 million (about $3.5 million) in 11 medical research projects that will investigate rare childhood diseases, including Dravet syndrome.

The GOSH Charity and Sparks National Call is a U.K. initiative to support medical research into pediatric rare diseases, as well as conditions that start in childhood. Grants are allocated to projects aiming to develop better therapies or diagnostics, or potential cures for rare disorders in children.

“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,” said Louise Parkes, chief executive at GOSH Charity, in a press release posted by the University of Manchester, whose project is among those chosen for support.

“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 … 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,” Parkes added.

The funding is the largest such charitable grant-giving program of its kind, the release states.

Sparks Charity, a children’s medical research charity that recently merged with GOSH, contributed a total of £900,000 (roughly $1.25 million). Acrodysostosis Support & Research, and Dravet Syndrome UK, two partner charities, provided another £112,500.

A project by researchers at the University of Oxford, titled “uORF-mediated SCN1A up-regulation for the treatment of Dravet syndrome,” will study a new potential treatment strategy for Dravet syndrome.

Other awarded projects include efforts into a new approach for testing potential treatments for acrodysostosis, a genetic disorder that affects bone growth, by researchers at UCL Great Ormond Street Institute of Child Health; better understanding of pain in children with cancer, aiming to improve their quality of life, by researchers at the University of Southampton; and developing a more effective treatment for Diamond-Blackfan Anaemia, a rare blood disorder, by researchers at University College London.

Researchers from the U.K. were invited to apply for funding as part of the GOSH Charity and Sparks’s National Call.