Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) will be an option for many more patients in the US after the FDA expanded the breadth of its approved labelling. Elevidys ...
The only other gene therapies to have made it to market so far are uniQure’s Glybera’s (alipogene tiparvovec), and GSK’s Strimvelis, but both have only gained EU approval. Glybera is a case ...
Some people live a full life with MD. Others, including those diagnosed with Duchenne muscular dystrophy (DMD), typically have lived into their teens and 20s. Muscular dystrophy, a genetic disease ...
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