muscular dystrophy
Patient Dies After Treatment With Sarepta's Duchenne Muscular Dystrophy Gene Therapy
The firm's stock price dropped 27 percent on Tuesday following the news that the young man died from acute liver failure after getting Elevidys.
The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did not meet the primary endpoint.
Belief BioMed's Duchenne Muscular Dystrophy Gene Therapy Gets FDA IND Clearance
The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.
Parents are suing the defendants for negligent genetic counseling and testing that they allege precluded their ability to avoid having children with Duchenne.
Sarepta Licenses Clinical, Preclinical siRNA Candidates From Arrowhead, Enters Discovery Deal
Sarepta will pay Arrowhead Pharmaceuticals $500 million upfront, make a $325 million equity investment, plus pay $250 million over five years, and more.