Researchers from University College London have jointly received a £1 million grant from LifeArc and Muscular Dystrophy UK, to develop potential treatments for congenital muscular dystrophy.

Congenital muscular dystrophy includes a group of conditions that can lead to muscle weakness, reduced movement and tight joints. Symptoms can start from birth, or in the first few months of life, and there are currently no effective treatments.

The first of the three research projects will use ‘mini muscles’ to find new treatments. Professor Francesco Saverio Tedesco aims to improve how potential treatments for LMNA-related congenital muscular dystrophy are tested, by growing ‘mini muscles’ in a dish that mirror the changes seen in people with LMNA. Using this method, Professor Tedesco hopes to identify the most promising treatments that can be taken to the next stage of research.

The second project will focus on improving the way that treatments are delivered to muscle cells. Professor Haiyan Zhou and team have already identified potential treatments for collagen VI-related muscular dystrophy. However, they have found it difficult to deliver the treatments to where they are needed in the body. The team have recently identified a targeting system which could help deliver the treatment. This project aims to link the targeting system with the potential treatment and test how well it works.

The third project will explore whether differences in DNA can protect muscles. Everyone has differences in their instruction manual – DNA – making them unique. Sometimes these differences can cause conditions such as congenital muscular dystrophy while, other times, these differences can help to protect the body from damage. Previous research has shown a specific DNA change may protect muscles in people with merosin-deficient congenital muscular dystrophy. In this project, Professor Francesco Muntoni will aim to design a treatment which can mirror this DNA change and potentially reduce the severity of the condition.

“LifeArc is committed to supporting research that is focused on patient impact, in underserved areas. Congenital muscular dystrophy includes a group of devastating conditions, for which there is no cure and limited treatment options. By funding these research projects, in partnership with Muscular Dystrophy UK, we aim to improve the lives of patients living with these conditions.”

Dr Joanna Davidge, Head of Funding at LifeArc

Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK, the leading charity for over 110,000 people in the UK living with one of over 60 muscle wasting and weakening conditions, adds: “With no effective treatments for congenital muscular dystrophy, there is a need to develop new treatments. We’re delighted to partner with LifeArc to fund groundbreaking research that brings us closer to potentially life-changing treatments for those living with a condition. This partnership was made possible thanks to an incredibly generous legacy.”

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