European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne Muscular Dystrophy (DMD)Designation is paving the way for safer long-term ...

Genethon is determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic ...

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...

Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

Sarepta continues to work with regulators to complete the safety label update for Elevidys, and they are discussing an approach for risk mitigation for non-ambulatory patients.

The FDA said it was clearing the way for Sarepta Therapeutics to resume shipments of its gene therapy for Duchenne muscular ...

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...

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