Davia Jones said it took seven years for her son, Kyrie, to be diagnosed with Duchenne Muscular Dystrophy, a progressive ...

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 ...

Sarepta Therapeutics (SRPT) stock jumps as the company posts early data from Phase 1/2 trials for its experimental muscular ...

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

Shares of Cambridge-based Sarepta rose about 35 percent in trading Wednesday after the company presented preliminary data on ...

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 ...

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 ...

Completed a US$57.2 million equity financing; commenced trading on the Nasdaq Global Market under the ticker “MSLE” on Feb. 6, 2026Secured global ...

Interim observations from the Phase 2 TRAILHEAD study show continued improvement in handgrip strength, overall stability of ...

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 ...

A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an experimental compound ...