Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), ...

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

Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in ...

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

Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio ...

Sarepta Therapeutics (NASDAQ:SRPT) reported a second fatality from acute liver failure in a patient treated with its gene ...

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

A new investigation into Duchenne muscular dystrophy (DMD) pathogenesis suggests that at least part of the muscle degeneration observed in DMD patients may result from the reduced production of ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...

This is certainly the case in Duchenne Muscular Dystrophy (DMD), a cruel, muscle-wasting condition, primarily affecting boys, which eventually robs them of their ability to walk, and drastically ...

Patients with myotonic dystrophy have smaller hearts, and lower systolic and diastolic blood pressures and pulse rates. They have impaired autonomic reflexes, measured by orthostatic, Valsalva ...