Biogen to Share New SMA Data at Muscular Dystrophy Association and SMA Europe Conferences

Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA)New Phase 1b data further illustrate potential of salanersen in SMA, ...
Healio

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

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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, ...

Nationwide MDA Shamrocks Campaign Advances Independence for People Living with Rare Neuromuscular Conditions

Thousands of retailers will participate with in-store MDA Shamrocks pin-ups and roundups throughout February and March. New limited-edition MDA Shamrocks T-shirt, designed by volunteer MDA Board ...

3K Run for Rare–2026 organised

Vijayawada: Marking World Rare Disease Day, the Amaravati Rare Diseases Organisation (ARDO) organised the “3K Run for Rare–2026” awareness programme on Friday at BRTS Road near Sarada College ...
Business Insider

Muscular Dystrophy Association Calls FDA Approval of Novartis’ Itvisma (onasemnogene abeparvovec-brve) a Major Step Forward for the Spinal Muscular Atrophy Community

New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed ...