日前，2026年肌营养不良协会（Muscular Dystrophy Association，MDA）临床与科学大会在美国奥兰多落下帷幕。MDA临床与科学大会是一项专注于神经肌肉疾病研究与临床进展的重要国际会议。在今年的大会上，多家生物医药公司分享了针对不同神经肌肉疾病创新疗法的最新进展。 寡核苷酸疗法作为一种靶向RNA的创新治疗模式，在治疗包括杜氏肌营养不良症（DMD）在内的多种神经肌肉疾病中已 ...

The big pharma company will no longer progress emugrobart to late-stage trials in FSHD and SMA due to a lack of efficacy.

Muscular dystrophies are a group of more than 30 genetic disorders characterized by progressive muscle weakness and wasting. These disorders are typically associated with defects in muscle ...

Skeletal muscle is a dynamic and highly adaptable tissue responsible for voluntary movement, posture and metabolic regulation. Its unique architecture, involving myofibres, satellite cells and a ...

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on sevasemten, an ...

While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Detailed price information for Tenaya Therapeutics Inc (TNYA-Q) from The Globe and Mail including charting and trades.

Duchenne muscular dystrophy (DMD) is a neuromuscular disease causing progressive skeletal muscle weakness and fatigue. The research team led by Associate Professor Hidetoshi Sakurai (Department of ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

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 ...