One million pound boost for neuromuscular research

Published Date
19/09/2014
Author
Neil Bennett
Category
Research

We are delighted to announce the investment of over one million pounds into a cutting-edge research programme, covering a number of conditions and potentially benefitting others, giving a dramatic boost to neuromuscular research.

Ten research projects, listed below, will be funded at a total cost of £1,086,264 over four years, a sum that has been made possible thanks to collaborations with other research charities that are co-funding several of the projects. These include a newly founded partnership with The SMA Trust and our ongoing partnership with the Duchenne Forum, a funding partnership of UK charities that supports a programme of world-class research to find effective treatments and cures for Duchenne muscular dystrophy, which was established last year.

The newly awarded projects cover nine different conditions, demonstrating our commitment to actively tackling a broad range of muscle-wasting conditions through our research programme. Over the next four years, the researchers working on these ten projects will address important research questions that will investigate the underlying biological mechanisms of muscle disease and test the feasibility of new potential treatments.

We are proud to be funding projects which offer the first steps to adapting ‘molecular patch’ or ‘exon-skipping’ technology, first developed for Duchenne muscular dystrophy, for spinal muscular atrophy (SMA), FSH and Ullrich muscular dystrophy. Two decades ago, when we began funding the development of exon-skipping, a treatment for Duchenne muscular dystrophy  must have seemed barely conceivable; today, potential treatments are in advanced clinical trials. We are delighted to also be funding pioneering research that could offer the building blocks for the next generation of potential treatments, broadening the pipeline for future therapies for muscle-wasting conditions.

The projects have been affirmed as ‘gold standard’ research by our Medical Research Committee, having been thoroughly reviewed by independent, international experts to verify their high quality, and selected with input from our Lay Research Panel to ensure the views of people affected by muscular dystrophy and related neuromuscular conditions are represented and that the research we fund is relevant to our supporters (read more about our selection process).

Professor David Abbott – Exploring the views of men with Duchenne muscular dystrophy on end-of-life care decision making

Professor Tom Gillingwater – Protecting motor neurones in spinal muscular atrophy

Professor Darren Monckton – Understanding the genetics of myotonic dystrophy type 1

Professor Jenny Morgan – Understanding muscle fibre death in Duchenne muscular dystrophy

Professor Francesco Muntoni – Developing a therapeutic approach for collagen VI-related muscular dystrophy

Professor Francesco Muntoni – Identifying biomarkers for congenital and limb girdle muscular dystrophies

Dr Andrew Shevchuk – Understanding cell membrane changes in centronuclear myopathy and Charcot-Marie-Tooth disease

Dr Saverio Tedesco – Developing a cell- and gene-based therapy for Duchenne muscular dystrophy

Dr Matthew Wood – Developing a genetic therapy for spinal muscular atrophy

Professor Peter Zammit – Generating a model of FSH and testing a potential therapeutic approach

It is only through your contributions that we can continue to fund the vital work that takes us closer to finding treatments and cures for muscle-wasting conditions. Donate now and help change the lives of thousands of people living with muscular dystrophy and related neuromuscular conditions. Thank-you for your support.

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