This project is jointly funded by Sparks and charity partner Great Ormond Street Hospital Children’s Charity through the National Research Funding Call.
Spinal muscular atrophy (SMA) is the most common genetic cause of death in infants in the UK. It is estimated that there are approximately 2,000 – 2,500 children and adults living with the condition in the UK.
SMA is a nerve- and muscle-wasting condition, sometimes classed as a form of motor neurone disease. There are several subtypes of SMA, but, sadly, there is no cure for any of them.
Recent research has led to the identification of several approaches that could help children with the most severe type of SMA, but it’s not clear whether the underlying causes of this form are shared by other types of SMA.
Dr Heidi Fuller and her team from Keele University want to understand more about the biology of the different types of SMA and identify new drugs that could help to treat those children.
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 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.
Paving the way towards a cure for nerve-wasting conditions (spinal muscular atrophy)
Researcher: Heidi Fuller