Up to three years of Evrysdi treatment appears safe and may help presymptomatic children with SMA reach and maintain key ...

Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases.

The president and CEO of the Muscular Dystrophy Association talks about what she's most excited about in neuromuscular ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

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 ...

(Reuters) -U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a muscular dystrophy patient who received a different, experimental ...

AI-ECG provides a non-invasive, accessible alternative for detecting LVSD in muscular dystrophy patients, overcoming challenges of routine echocardiography. The study showed AI-ECG's high sensitivity ...

FDA evaluating further regulatory actions after deaths Elevidys is the only gene therapy for Duchenne muscular dystrophy Sarepta suggested updating Elevidys' label June 24 (Reuters) - The U.S. Food ...

On Sunday at 1 a.m. Eastern time, an announcement went out from the first-generation gene therapy company for Duchenne muscular dystrophy: A second patient who had been treated with Elevidys has died.