Sarepta Therapeutics said it has temporarily suspended shipments of Elevidys® (delandistrogene moxeparvovec-rokl) for infusion in non-ambulatory patients following the second death in three months of ...

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

Therapeutics provided a safety update regarding Elevidys, an FDA approved gene therapy for patients with Duchenne muscular ...

Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for a rare muscle disorder.

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 ...

The campaign to commemorate the work of the Muscular Dystrophy Association was created in partnership with agency Yes&.

As diagnoses of autism spectrum disorder rise, a UNLV researcher's breakthrough could lead to new forms of treatment ...