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

Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the company over a mounting string of controversies—to the FDA, Endpoints News reported Thursday, citing an unnamed senior FDA official.

Sarepta stock plunged again Thursday on a report the FDA will require additional clinical testing to validate the safety profile of Elevidys.

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.

Sarepta Therapeutics (SRPT) stock drops as the company faces potential FDA-mandated studies after safety concerns over its gene therapy, Elevidys. Read more here.

The family of a seven-year-old boy diagnosed with a life-changing condition are raising funds to build a specially-adapted home.

A 15-year-old boy in New Market with a neuromuscular disorder received a wheelchair accessible van last week through the support of family, community and a nonprofit dedicated to assisting those

The Food and Drug Administration won’t sign off on Sarepta Therapeutics Inc. bringing its controversial gene therapy back to the market until the company can persuade US regulators that it won’t cause more deaths,

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need,