Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular dystrophy over safety issues, even as patients and investors expressed growing concern about the company’s decision-making.

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments.

Children’s Hospital Los Angeles paused the use of Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy in the wake of rising safety concerns about liver toxicity, adding to mounting challenges for the drugmaker.

ICER is listening to concerns like McSherry's, and Pearson says feedback from the Duchenne patient community is being incorporated into the organization's work.

Shares of Sarepta Therapeutics (NASDAQ: SRPT) plunged 26.85% in Friday pre-market after a clinical trial participant died following treatment with a gene therapy, marking the third death linked to the company's experimental treatments.

Finally, it seemed, Sarah Jenssen would have a chance to get a treatment that her family hoped could help her. A gene therapy for Sarah’s condition, Duchenne muscular dystrophy, won a wide ...

“With the benefit of newborn screening, the next generation of families will have even more time to evaluate, pursue and access FDA-approved Duchenne therapies while avoiding extensive surgeries ...