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DMD gene therapy Elevidys linked to new liver failure case
Liver damage is a well-recognised complication of gene therapies based on adeno-associated virus (AAV) vectors, and other drugs – including Novartis' spinal muscular atrophy (SMA) treatment Zolgensma (onasemnogene abeparvovec) and Astellas' X-linked myotubular myopathy therapy AT132 – have been associated with deaths due to acute liver failure.
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Sarepta and Roche halt DMD gene therapy’s use after second deathShares in both companies were down following news of the second death, casting fresh safety concerns over Elevidys.
For the second time, a person with DMD has died of acute liver failure after being treated with the one-time gene therapy Elevidys.
Sarepta Therapeutics (NASDAQ:SRPT) fell ~38% in the premarket on Monday after the company reported a second fatality from ...
Following a second patient death, Sarepta is temporarily suspending use of Elevidys in non-ambulatory DMD patients, pending ...
Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory ...
A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile. | A second patient has ...
Sarepta suspends Elevidys use in non-ambulatory Duchenne patients after two liver failure deaths, triggering FDA review and ...
Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain ...
Sarepta Therapeutics Inc. plummeted after reporting a second patient died of acute liver failure while being treated with its ...
BASEL, Switzerland I June 15, 2025 I Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ ...
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