As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...

The move follows the death of two teenagers who were administered the treatment, as well as the death of a 51-year-old ...

Elevidys is a gene therapy approved to treat Duchenne muscular dystrophy. Last month, Sarapta halted sales of the therapy for non-ambulatory patients.

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

The FDA called for Sarepta Therapeutics to stop shipping its muscular dystrophy gene therapy, but the company said no.

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

Sarepta Therapeutics lays off 493 workers amid FDA probe, stock drop, and concerns over its gene therapy treatment, Elevidys.

The company had previously halted shipments to certain older patients following the deaths of two teenagers treated with the ...

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

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

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...

On a conference call, CEO Doug Ingram said the decision to cut 36% of the company's staff and halt several drug programs was ...