gene therapy, muscular dystrophy
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Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other parents still push for the therapy.
A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS
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Why Sarepta Therapeutics (SRPT) Stock Is Down TodayShares of biotech company Sarepta Therapeutics (NASDAQ:SRPT) fell 44.4% in the afternoon session after the company reported a second death linked to its Duchenne muscular dystrophy gene therapy. Elevidys.
Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory patients with Duchenne muscular dystrophy following two deaths caused by acute liver failure.
The commercial and clinical use of Elevidys was discontinued after two fatal cases of acute liver failure.