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Sarepta stands behind Elevidys after FDA requests gene therapy be pulled from market
As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm behind its approved Duchenne muscular dystrophy (DMD) treatment. | As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head,
A Cambridge-based company developing gene therapies for rare diseases is laying off more than a third of its workforce.
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...
Melanie Sanford fought to get her son Hudson a breakthrough gene therapy to stop the progression of the fatal disease Duchenne Muscular Dystrophy. She rushed to get him access just days ...
The FDA expanded the approval of delandistrogene moxeparvovec-rokl (Elevidys) gene therapy for Duchenne muscular dystrophy on Thursday to include ambulatory or non-ambulatory patients ages 4 ...
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