Roche discontinued the commercial and clinical use of Elevidys after two cases of fatal acute liver failure. The Swiss ...

Despite setbacks, Sarepta’s gene therapy pipeline and market leadership in DMD present a high-risk, high-reward setup. Read ...

Retail investor chatter around Sarepta Therapeutics surged on Sunday after the company announced it had suspended shipments ...

Elevidys is the only gene therapy approved by U.S. drug regulators for Duchenne muscular dystrophy in patients aged four and above. "The second patient death may raise serious questions on Elevidys' ...

Sarepta Therapeutics said it has temporarily suspended shipments of Elevidys® (delandistrogene moxeparvovec-rokl) for ...

Sarepta Therapeutics ( NASDAQ: SRPT) reported a second fatality from acute liver failure in a patient treated with its gene ...

Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death ...

Sarepta Therapeutics on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy for a rare form of muscular ...

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) ...

PepGen Inc. overstated prospects for its leading product candidate to treat a muscle-degenerating genetic disorder before facing negative responses to its clinical trial data and ultimately stopping ...

Solve FSHD and Modalis Therapeutics Corp. have established a strategic collaboration to develop an innovative therapy for facioscapulohumeral muscular dystrophy (FSHD).

Avidity has been given the go-ahead by the FDA to request accelerated approval of delpacibart braxlosiran—potentially the ...