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FDA takes U-turn on Sarepta’s Elevidys, backing Duchenne gene therapy again in ambulatory patients
On Monday, the FDA said Sarepta may resume treating ambulatory Duchenne patients with Elevidys. The recommendation comes just 10 days after the agency requested that the company halt all shipments of the one-time treatment following reports of three deaths among patients who had received a Sarepta gene therapy, including two on Elevidys.
The Duchenne muscular dystrophy market is witnessing steady growth driven by advancements in gene therapies, exon-skipping drugs, and corticosteroid alternatives. Increasing diagnosis rates, rising ...
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 ...
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...
European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne ...
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Roche Bets Big On Alzheimer’s Prevention With New Trial As Duchenne Drug Setback LoomsRoche Holdings AG said it will begin a late-stage trial to evaluate whether its experimental Alzheimer’s drug, trontinemab, ...
Genethon is determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic ...
Basel: Roche has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency ...
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