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

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 ...

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 ...

A successful Phase 3 trial and a public offeringof stock boosted prospects for this company to receive approval for an ...

—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 ...

(Reuters) - The U.S. FDA has approved privately held Italfarmaco Group's drug to treat Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disorder, the health regulator said on Thursday.

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, ...

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 ...

Southeast Texas WhiskeyFest proceeds will support the Cure Rare Disease nonprofit, which has become a life mission for Mike ...

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, ...