A second case of acute liver failure (ALF) resulting in the death has been reported in a young recipient of Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy.
The cases of ALF to date have both occurred in non-ambulatory patients with Duchenne.
Shares in Sarepta Therapeutics (Nasdaq: SRPT) were nearly 50% lower early Monday, while those in partner Roche (ROG: SIX) were 2% lower, as markets digested the news.
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