Muscular Dystrophy UK: A £1 million partnership to find new treatments for congenital muscular dystrophy
We’ve partnered with Muscular Dystrophy UK to fund promising research at University College London (UCL) that aims to accelerate the development of much-needed treatments for congenital muscular dystrophy.
Overview
Our joint £1 million in funding supports researchers at UCL to advance innovative approaches to treating congenital muscular dystrophy—a group of rare, muscle-wasting conditions that start in infancy and currently have no effective treatments. The funding supports 3 separate research projects focused on identifying and developing targeted therapies.
About the partnership
The projects receiving funding each explore different approaches that could help support the development of treatments. These include:
Using ‘mini muscles’ to improve treatment discovery
Led by Professor Francesco Saverio Tedesco, this project will create lab-grown muscle tissue— ‘mini muscles’—that mimic the disease characteristics of LMNA-related congenital muscular dystrophy. These models will allow researchers to more accurately test potential therapies and identify the most promising candidates to take forward into further development.
Improving delivery of treatments to muscle cells
Professor Haiyan Zhou and her team are building on their previous work identifying potential therapies for collagen VI-related muscular dystrophy. This project will focus on developing a delivery system that ensures these treatments reach the muscle cells effectively. The team plans to link the therapeutic compound to a targeting system they recently discovered and assess how well it improves drug delivery in preclinical models.
Mimicking protective DNA changes to reduce disease severity
Professor Francesco Muntoni will explore how a specific DNA variation—found in some individuals with merosin-deficient congenital muscular dystrophy—may naturally protect muscles from damage. The goal is to design a treatment that mimics this protective change, potentially reducing the severity of symptoms in patients with this subtype of the condition.
About congenital muscular dystrophy
Congenital muscular dystrophy refers to a group of rare genetic conditions that cause muscle weakness, tight joints, and limited mobility. Symptoms typically appear at birth or in the first few months of life. The condition can severely impact a child’s quality of life, and there are currently no effective treatments or cures. This leaves families facing a future of uncertainty and limited medical options.
Impact of the partnership
This research partnership has the potential to transform the future for children and families affected by congenital muscular dystrophy. By focusing on novel delivery systems, improved disease modeling, and protective genetic mechanisms, these projects could pave the way for entirely new classes of treatments. The long-term goal is to bring life-changing and possibly life-saving therapies closer to the clinic, offering hope to a community that has waited far too long for breakthroughs.
“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.”
Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK
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