A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

A mere 5 months ago, our world turned upside down when our grandson William was diagnosed with Duchenne muscular dystrophy (DMD). We had been searching for answers, worried about his struggles with ...

Spinal muscular atrophy (SMA) is a disorder affecting the motor ... SMA type 3 (juvenile SMA, Kugelberg Welander syndrome): Type 3 SMA emerges in children 18 months old or older and can become evident ...

Becker muscular dystrophy (BMD) is a type of muscular dystrophy, a genetic condition that weakens and damages muscles. It can worsen with age. However, its symptoms are less severe than those of ...

Credit: Shutterstock / ChiccoDodiFC. The beginning to the year has been eventful in the Duchenne muscular dystrophy (DMD) space, with several trial readouts announced at the Muscular Dystrophy ...

Epicrispr Biotechnologies has raised $68 million in pursuit of a first-of-its-kind genetic medicine for a rare neuromuscular disorder called facioscapulohumeral muscular dystrophy. EPI-321, the ...

Thymidine kinase 2 deficiency is an ultra-rare, life-threatening, genetic mitochondrial disease characterized by progressive and severe muscle weakness (myopathy), which can impact the ability to walk ...

Brussels (Belgium) 19 March 2025 – 07:00 AM (CET) – UCB, a global biopharmaceutical company, today announced positive data from studies involving its investigational pyrimidine nucleoside therapy, ...

Myotonic dystrophy type 1 incurs high healthcare costs, driven by age and comorbidities like cardiac and respiratory issues. Early detection and integrated management of multisystemic ...

A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%.