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

The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...

A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to the company's gene therapies ...

Second patient death from liver failure after Sarepta's Elevidys gene therapy triggers FDA investigation. Stock crashes 41% ...

Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its ...

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

Patients with myotonic muscular dystrophy are at increased risk for cancer of the brain, ovary, colon, and the uterine lining known as the endometrium. Skip to main content.

In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and ...

Complex, multi-system diseases like myotonic dystrophy – the most common adult form of muscular dystrophy – require physicians and patients to identify which symptoms impact quality of life ...

Children's Hospital Los Angeles paused Sarepta's Elevidys gene therapy after FDA flagged safety issues, including deaths in ...

Sarepta's muscular dystrophy treatment is the first gene therapy approved under the program. The disease, which almost exclusively affects boys, destroys muscles. Most boys end up in wheelchairs ...

(Reuters) -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 ...