New York, Jan. 26, 2026 (GLOBE NEWSWIRE) -- In recognition of Rare Disease Day on February 28, the Muscular Dystrophy Association (MDA) is sharing powerful community stories throughout the month that ...
The MarketWatch News Department was not involved in the creation of this content. New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and ...
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 ...
Newly approved SMA therapies show promise but face global accessibility challenges, widening healthcare disparities due to high costs and limited availability. Newborn screening for SMA is widespread ...
New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of individuals with Types 2 or 3 spinal muscular atrophy (SMA) Late-breaking ...
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...
Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...
With a tank of zebrafish, Queensland researcher Jean Giacomotto was able to test a never-before seen genetic variant ...
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 ...
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