New report published on newborn screening for rare conditions
Muscular Dystrophy UK and the All-Party Parliamentary Group for Muscular Dystrophy publish a new report on newborn screening for rare conditions.
US FDA advisory committee votes on Sarepta SRP-9001 gene therapy to treat Duchenne muscular dystrophy ahead of a FDA’s final decision
Last week an advisory committee for the US Food and Drug Administration (FDA) met to discuss Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy.
Ways you can get involved
For over 50 years, we've been here for people living with muscle-wasting conditions. We couldn't do this without your help. Thank you.
Support for our work is more crucial than ever, so we've got lots of ideas about how you can help us fundraise.
Our brilliant volunteers help us deliver services, fundraise and raise awareness about muscle-wasting conditions.