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Latest news
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.
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