At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

The drastic cost-cutting move follows the deaths of two teenagers that forced the company to restrict usage of its gene ...

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...

Sarepta Therapeutics is facing a challenging period after the deaths of two patients who received its Columbus-invented gene ...

Sarepta Therapeutics appears to have right-sized itself after laying off over a third of its staff, announcing a significant ...

The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment ...

Following the death of two teenage patients with Duchenne muscular dystrophy following Elevidys treatment, Sarepta ...

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...

FDA issued Complete Response Letter Capricor plans to resubmit its BLA to include data from the ongoing Phase 3 HOPE-3 trial ...

Sarepta Therapeutics said it will eliminate 36% of its workforce—approximately 500 jobs—in a restructuring that follows the ...

The company is also pausing research for several treatments it has been developing for another form of muscular dystrophy.