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 ...

Outperformed Approved Pan-HDAC Inhibitor Givinostat in Improving Muscle Function and Correcting Drivers of DMD Cardiomyopathy ...

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 ...

Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients ...

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 ...

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today presented at the 2026 ...

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 ...

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases ...

Brooklyn Nets radio play-by-play voice Chris Carrino Sr. was honored by the Naismith Basketball Hall of Fame with a Curt Gowdy Media Award.

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 ...

Longboat Key firefighters “filled the boot” through a fundraising effort for the Muscular Dystrophy Association.