Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...

A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys ...

Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...

Sarepta Therapeutics ( NASDAQ: SRPT) fell ~38% in the premarket on Monday after the company reported a second fatality from ...

Shares of biotech company Sarepta Therapeutics (NASDAQ:SRPT) fell 44.4% in the afternoon session after the company reported a ...

The firm will stop shipping the gene therapy for these patients until a new immunosuppressive regimen is approved for managing acute liver failure.

Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for ...

Key Takeaways Sarepta Therapeutics shares sank to their lowest level in nine years Monday after a second patient taking its ...

Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain patients while it evaluates new safety precautions.

Liver damage is a well-recognised complication of gene therapies based on adeno-associated virus (AAV) vectors, and other ...