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 ...

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 ...

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

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single ...

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 ...

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...

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 ...

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.