WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the ...

Givinostat will be made available to eligible patients with Duchenne muscular dystrophy after its manufacturer struck a ...

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 ...

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 ...

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 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) ...

There’s news of a potential treatment that may help patients with muscular dystrophy and it’s giving hope to those with a debilitating genetic disease. There are more than 50 forms, but not one ...

WASHINGTON — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting in morning trading. The young ...