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Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU
EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...
The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.
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 ...
Sarepta Therapeutics suffered another major setback on Friday as Europe's drug regulator decided not to recommend the ...
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