What we do
Muscular dystrophy and related neuromuscular conditions are regarded as rare and, in many cases, even ultra-rare conditions. There are currently no efficient treatments or cures available for most of these conditions. Research, however, advances fast and a number of clinical trials to test the clinical benefit of promising technology are under way.
Over more than 50 years, the charity has contributed to crucial scientific breakthroughs in the field of muscular dystrophy and related neuromuscular conditions such as: laying the foundations for the promising technology of 'exon skipping' which is currently being tested in clinical trials for boys with Duchenne muscular dystrophy, and funding work that has led to a scientific breakthrough in finding a treatment for mitochondrial myopathy, which is now close to clinical trial.
Scientists and clinicians are beginning to express cautious optimism that treatments might become a reality in the near future, but to do this we need to continue to fund research. We aim to find treatments and ultimately cures for muscular dystrophy and related neuromuscular conditions and improve the lives of everyone affected by them and we need your help to do this.
Our work has five main focuses:
- we fund world-class research to find effective treatments and cures
- we provide practical information, advice and emotional support for individuals with muscular dystrophy and other related conditions, their carers and families
- we campaign to bring about change and raise awareness of muscular dystrophy and other related conditions
- we award grants towards the cost of specialist equipment, such as powered wheelchairs
- we provide specialist education and development for health professionals.