Jake Kirchner wanted to play baseball in Belle Plaine. This spring, he is, in a way. Mike Max explains.

A MUM who was told that her toddler’s waddle was normal later discovered he had a “life limiting” condition that will stop ...

PBGENE-DMD aims to deliver sustained muscle function improvement for DMD patients with mutations between exons 45 and 55.

Nomia with her daughter Maham who has to be specially ventilated for two hours during the day as well as all night The family ...

The Phase 1/2 FUNCTION-DMD study will enroll ambulatory patients and is designed to evaluate safety, tolerability, and efficacy, including dystrophin protein expression and functional outcomes. "The ...

Jon Anning, a Slippery Rock University professor, has done extensive research on effects and treatment for Duchenne muscular ...

The EMA has backed broader pediatric use of Agamree and Crysvita, lowering age thresholds for rare genetic diseases affecting muscle and bone development.

Santhera Pharmaceuticals (SIX: SANN) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending ...

Deramiocel is a novel cellular therapy that demonstrated benefit on skeletal muscle function in the HOPE-2 phase II study of ...

Alan Osmond was first diagnosed with multiple sclerosis (MS) in the late 1980s. Osmond spoke with PEOPLE about his experience ...

Learn how to exercise safely with Duchenne muscular dystrophy, for your flexibility, mobility, and overall health, without ...

Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced the presentation of data from its Phase 3 ...