Vamorolone is approved and marketed as AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD) in the US, European Union, UK and ChinaNew ...

Swiss specialty drugmaker Santhera Pharmaceuticals announces it has signed an exclusive licensing agreement with Nxera Pharma ...

WASHINGTON, April 23, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization, and the UK charity Duchenne UK, two leading organizations dedicated to ending Duchenne ...

—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 ...

Duchenne muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each ...

The Duchenne muscular dystrophy drugs now available only treat the symptoms of the rare muscle-wasting disorder. On Thursday, the FDA approved a Sarepta Therapeutics gene therapy, making it the first ...

Interim data showed 110% average microdystrophin expression and up to 88% dystrophin-positive muscle fibers in the first three participants. Two patients saw an 8% average heart function improvement ...

Tracing the impact of a single protein, Piezo1, researchers found that restoring it in muscles affected by Duchenne muscular dystrophy could improve their ability to heal efficiently. One protein, ...

A Community Event for Duchenne Muscular Dystrophy Awareness Brick, United States – January 1, 2026 / Steps for Hope 5K / The inaugural Steps For Hope 5K is set to take place in Brick, New Jersey, ...

Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular atrophy. Researchers have ...