Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease progression and showed sustained benefit ...
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 ...
Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology and functional decline in a mouse model of facioscapulohumeral muscular ...
The U.S. Food and Drug Administration has awarded orphan drug status to Atossa Therapeutics' (Z)-endoxifen for Duchenne ...
Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE ...
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