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 ...

A new study looked at the electronic medical records of 68 patients treated for DMD at a tertiary referral center. Most patients—approximately 90%—were taking angiotensin-converting enzyme (ACE) ...

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 ...

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 ...

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 ...

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

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 ...

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 ...