Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, and Advocacy

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...
Healio

Nusinersen linked to improved motor function in spinal muscular atrophy

Please provide your email address to receive an email when new articles are posted on . Interim results also showed reduction of the neurodegenerative biomarker neurofilament light chain. Serious ...
Healio

Gene therapy improves motor function in spinal muscular atrophy at 52 weeks

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Patients treated with onasemnogene abeparvovec recorded ...
Business Wire

Scholar Rock to Present Additional Clinical Data from the Phase 3 SAPPHIRE Trial at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...
News Medical

Key gene found to prevent muscle breakdown in muscular dystrophy

A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular dystrophies. This is shown in a new study at Umeå ...
FierceBiotech

Roche and Abu Dhabi government to collect data on rare spinal diseases

Roche and the government of Abu Dhabi have set up a new public-private partnership to study spinal muscular atrophy and Duchenne muscular dystrophy (DMD) in the Middle East. In a May 21 announcement, ...