Keele University’s Dr Heidi Fuller has received a grant from Great Ormond Street Hospital Children’s Charity (GOSH) and Sparks, the medical research charity, to fund a study that will help pave the way towards a cure for the muscle-wasting condition spinal muscular atrophy (SMA).

This is part of a £2.1 million investment by the two charities - the largest charitable funding call dedicated to child health research in the UK.

Dr Fuller hopes that her work will lead to new clinical trials for different types of SMA, and help understand if treatments for the most severe type of the condition could be used in children with less severe forms. This could lead to new and more effective treatments for this devastating condition and pave the way for a cure.

Dr Heidi Fuller said:

“I am delighted to have received funding from the Great Ormond Street Hospital Charity and Sparks which will enable me to further my work.

“There is no cure for SMA but research progress has been made recently, leading to the identification of several approaches. All this work, however, has focused on identifying therapies that show promise for babies with Type I SMA - the most severe form. It has been assumed that the molecular defects in severe SMA are the same across other types of the condition.

“During this project, we plan to identify treatment strategies that are likely to be effective for children with Type II and III SMA. It will also help us to understand whether the treatment strategies that are being developed for babies with severe SMA will likely be effective for children with the other types of the condition.”

Tim Johnson, Chief Executive of GOSH Charity and Sparks said: “For many seriously ill children, research is their only hope, yet paediatric research is severely underfunded, receiving only five per cent of public and charitable funding research in the UK each year.

“By making more money available to researchers from across the country we will help them to find new ways to diagnose, treat and cure complex diseases that affect children.”

Kiki Syrad, Director of Grants and Impact at Great Ormond Street Hospital Children’s Charity said:

“The invitation to researchers to apply for funding received a huge response from the paediatric research community. We look forward to seeing how Heidi’s project progresses.”

 

---