Consensus directed medical therapy (CDMT) was used in 73.1% of DMD patients with moderate or severe left ventricular dysfunction, leaving nearly 30% without recommended treatment. Target doses were ...

Scotland’s NHS can now offer givinostat to ambulant children with Duchenne muscular dystrophy, marking a step forward in care ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. A second person with Duchenne muscular dystrophy has ...

In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and is an ...

—Surveys of caregivers and health care providers reveal gaps in screening and management of neuropsychiatric and neurodevelopmental symptoms among patients with Duchenne muscular dystrophy in the ...

Solid Biosciences Inc. (NASDAQ:SLDB) is one of the stocks that will double in 2026. On December 4, Needham analyst Gil Blum ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%. The young ...

A cell therapy stabilizes weakened muscles -- including the heart muscle -- in Duchenne muscular dystrophy patients, a new study shows. A cell therapy developed by the executive director of the Smidt ...

Like most mothers, I only saw perfection when my first son was born. That quickly changed when we began to question pediatricians about his physical and cognitive delays. Don’t worry, they said. Boys ...

A new study from UT Southwestern suggests that more people with Duchenne muscular dystrophy could live longer by identifying and more aggressively treating patients with certain risk factors. The ...

Please provide your email address to receive an email when new articles are posted on . Viltepso, an injection treatment for Duchenne muscular dystrophy in patients with a confirmed mutation, ...

Health secretary Robert F. Kennedy Jr. on Tuesday added two deadly rare diseases, Duchenne muscular dystrophy and ...