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 ...
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 ...
US FDA grants Orphan Drug Designation to Atossa Therapeutics’ (Z)-endoxifen to treat Duchenne muscular dystrophy: Seattle Monday, January 19, 2026, 17:00 Hrs [IST] Atossa Therap ...
Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...
Designation further supports (Z)-Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE ...
Muscular dystrophy is a genetic disorder that weakens muscles over time. There are many different types of muscular dystrophies. Each type begins at a different age and may cause mild or severe muscle ...
Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa" or the "Company"), a clinical-stage biopharmaceutical company developing novel therapies in oncology and other areas of high unmet clinical need, ...
Company on track to report ELEVATE-44-201 data from the first cohort in Q2 2026 and ELEVATE-45-201 data from the first cohort in mid-2026 - ...
Objective: To investigate the clinical manifestations, diagnosis and treatment, and DMPK gene mutations in neonates with congenital myotonic dystrophy (CDM). Methods: A retrospective analysis was ...
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