Latest news NICE publishes final guidance recommending access to Duchenne muscular dystrophy treatment Translarna Read more 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. See all news Ways you can get involved Donate 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. Find out more Fundraise Support for our work is more crucial than ever, so we've got lots of ideas about how you can help us fundraise. Find out more Volunteer Our brilliant volunteers help us deliver services, fundraise and raise awareness about muscle-wasting conditions. Find out more
NICE publishes final guidance recommending access to Duchenne muscular dystrophy treatment Translarna Read more
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.
Donate 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. Find out more
Fundraise Support for our work is more crucial than ever, so we've got lots of ideas about how you can help us fundraise. Find out more
Volunteer Our brilliant volunteers help us deliver services, fundraise and raise awareness about muscle-wasting conditions. Find out more