Research into Duchenne muscular dystrophy has gathered pace over the last few years, with Action Duchenne supporting and funding a number of clinical trials. More can be found out about research in research basics.

There are a number of new investigational compounds and drugs entering the late stage of the clinical trial process, with the recent approval of translarna to treat Duchenne caused by nonsense mutations (around 13-15% of cases).  

Clinical trials are an important part of medical research comparing the effects of a potential treatment with another treatment or placebo (drug with no effect). Clinical trials can either involve patients or healthy volunteers.

Find out more about  the recommendations and funding priorities of Action Duchenne for potential drug therapies in our Research strategy.

Your child can be signed up to the DMD registry to ensure they can be put forward for any suitable and relevant clinical trials.