Muscular Dystrophy UK has announced that it is investing £1.7 million into 12 new research projects across England and Scotland, including five at University College London (UCL).
The leading charity for more than 110,000 children and adults in the UK living with one of over 60 muscle wasting and weakening conditions is funding the research projects that will explore several different conditions including limb-girdle muscular dystrophies, laminopathies, Duchenne muscular dystrophy. For the first time in the charity’s history, it will fund research into CMT-SORD and periodic paralysis.
The charity funds research projects that aim to improve diagnosis, monitor progression, and test potential new treatments. The 12 new grants bring the total number of research projects funded by Muscular Dystrophy UK to 51.
Patrizia Ferretti, Professor of Regenerative Biology at UCL, who received a grant said: “Development of specific mechanism-based therapies for people living with Duchenne muscular dystrophy targeting emotional, cognitive, and behavioural problems could really make a big difference, by reducing anxiety and hyperactivity, and improving working memory. This could be beneficial by helping them settle in a school or work environment and in general social interactions. Overall, this would help to improve the quality of life, which would have positive outcomes for the individuals and their parents or carers supporting them.”
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Dr James Sleigh and team at University College London receive research grant from Muscular Dystrophy UK – Credit @ Muscular Dystrophy UK
Dr James Sleigh, Principal Research Fellow at UCL, who also received a grant from the charity said: “Our work will explore new models of genetic peripheral neuropathy, which is essential to help us better understand why motor and sensory nerves are selectively impacted by CMT/SORD neuropathy.
“The new model for SORD neuropathy will provide a key tool for testing therapeutic strategies that will hopefully one day help people living with the condition. This project will allow us to identify, track and correlate clinical variables in SORD neuropathy patients, which is a key step towards being able to evaluate condition progression and treatment success in the clinic.”
The charity will also be funding Henry Houlden’s project which will use state-of-the-art genetic sequencing techniques to find genes that cause limb-girdle muscular dystrophies. Dr. Roope Mannikko’s project aims to develop a new gene therapy for periodic paralysis, and Professor Francesco Saverio Tedesco’s project, at UCL and the Francis Crick Institute, will explore the link between the shape of nuclei and DNA organisation in muscle cells from people living with laminopathy.
Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK said: “Every year at Muscular Dystrophy UK, we look to fund the highest quality research that will not only bring research forward but also improve the lives of people living with muscle wasting and weakening conditions.
“We are always delighted to fund research into a variety of conditions including the five projects at University College London. We’ve seen an increasing number of conditions in our research portfolio including funding research into periodic paralysis and CMT/SORD neuropathy for the first time.”
As well as funding research, the charity also shares expert advice and support for the muscle wasting community to live well now, works with the NHS towards universal access to specialist healthcare and campaigns for people’s rights, better understanding, accessibility, and access to treatments.
