Healio

Apitegromab linked to improved outcomes for those with spinal muscular atrophy

Please provide your email address to receive an email when new articles are posted on . The study included 58 individuals with SMA types 2 and 3 who were treated with IV apitegromab for 52 weeks. At ...
Business Insider

FDA Approves New Tablet Form of Evrysdi for Treatment of Spinal Muscular Atrophy

New York, Feb. 12, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today celebrates the decision by the US Food and Drug Administration (FDA) to grant approval of a risdiplam ...

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, ...
Yahoo

Jesy Nelson Reveals Twins' Rare Diagnosis—What Parents Need To Know About Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) Type 1 is a rare but serious genetic condition that weakens muscles and can make basic activities like eating and breathing hard for babies. Early treatment—especially ...
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 ...
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, ...