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

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

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 | ...

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 ...

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

(Z)-Endoxifen is Atossa's investigational ER-modulating small molecule. In oncology and CNS studies to date, (Z)-endoxifen has shown a favorable safety profile and pharmacology distinct from tamoxifen ...

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

The U.S. Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...

Families caring for individuals with Duchenne muscular dystrophy (DMD) face significant financial burdens from necessary home and vehicle modifications to enhance quality of life. The Momentum trial ...

—Older age, presence of scoliosis, and other factors were associated with lower predicted forced vital capacity in patients with Duchenne muscular dystrophy using non-invasive ventilation. Individuals ...

When you're diagnosing a child with Duchenne muscular dystrophy, says Alexandra Bonner, MD, of Cleveland Clinic, it's important to not only determine if she's a carrier of the gene mutation that ...