Upsher-Smith Laboratories, LLC (Upsher-Smith) today announced the launch of KYMBEE™ (deflazacort) Tablets, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age ...

A 675km cycling challenge aims to raise funds for a Cumbrian children's hospice. Steve Alecock took on the challenge in ...

STATEN ISLAND, N.Y. — In the “Big Heart” department, Mike DiLeo, executive chef at Max’s Esca, played host to “Savor the ...

I’ve been reflecting on the disappointing outcomes and the reaction to Sarepta Therapeutics’ recently announced clinical ...

In July, the FDA refused to approve Capricor’s application for Deramiocel for the treatment of cardiomyopathy associated with ...

More than 25 Palm Beach County firefighters are training for the Palm Beaches Marathon to raise awareness and support for ...

MONDAY, Nov. 17, 2025 (HealthDay News) — The U.S. Food and Drug Administration (FDA) is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died.

The US FDA has tightened the use of a Duchenne muscular dystrophy gene therapy after two teenage patients died from acute liver failure following treatment. The agency has now restricted eligibility, ...

Testing the limits of his power chair’s off-road capabilities, 21-year-old Evan Procko parks on nature trails in some of the ...

When the FDA reworked the prescribing information for Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) gene therapy Elevidys earlier this month, the company touted a plan to study a regimen | ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients.