CHMP upheld earlier opinions that Translarna is not of significant benefit to DMD due to nonsense mutations; European ...

"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne ...

研究结果为DMD发病机制，特别是疾病早期的分子和细胞变化提供了新的见解。揭示了免疫、纤维化以及肌肉干细胞在DMD早期的动态变化，为早期干预 ...

The importance of family and community support took on a new meaning when our son Michael was diagnosed with Duchenne muscular dystrophy (DMD), a rare and severe neuromuscular disease. We started ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

(NASDAQ:CAPR) announced today that it intends to file a Biologics License Application [BLA] for deramiocel for the treatment of patients with Duchenne Muscular Dystrophy [DMD] cardiomyopathy.

Moving drugs from the lab bench to pharmacy shelves is no small task. In this article we delve into the role regulatory ...

Wave Life Sciences has met its goal in a Duchenne muscular dystrophy (DMD) study, positioning it to talk to regulators about accelerated approval while continuing to track patients through to the ...

DMD is a genetic disease that causes progressive muscle weakness and degeneration. Cardiac and orthopedic complications, including scoliosis and contractures (tightened muscles) The data was ...

dystrophin proteins are reduced or absent in DMD patients due to mutations in the respective gene. Symptoms such as muscle weakness and wasting present in the first few years of life with most ...

soared 51% Tuesday after the company reported positive interim data from a mid-stage trial of a treatment for the rare muscle disorder Duchenne muscular dystrophy. That put the stock on track for ...