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

The patient, who was being treated with an investigational gene therapy for limb-girdle muscular dystrophy, died of acute ...

Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...

MicroRNA-33a and microRNA-33b are encoded within the mammalian SREBF1 and SRBF2 genes, respectively, and both SREBF1 and ...

Sarepta plummets after a third death in its muscular dystrophy program for investigational gene therapies, prompts FDA action and intensifies safety scrutiny.

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

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

Children's Hospital Los Angeles, citing recent U.S. Food and Drug Administration actions, on Monday said it has paused usage of Sarepta Therapeutics' gene therapy Elevidys in all patients with ...

The regulator had asked Sarepta Therapeutics to halt all shipments of its therapy, Elevidys, after three patients died from ...

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

In a highly unusual move, Cambridge-based Sarepta said late Friday won’t comply with a request from the Food and Drug ...