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Dyne Therapeutics Announces MHLW In Japan Grants Orphan Drug Designation For Zeleciment Basivarsen For Treatment Of Myotonic Muscular Dystrophy Type 1

Benzinga·01/20/2026 12:31:56
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- Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE clinical trial -

- Expect to complete enrollment in ACHIEVE Registrational Expansion Cohort in early Q2 2026 -

WALTHAM, Mass., Jan. 20, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for zeleciment basivarsen (z-basivarsen), for the treatment of myotonic muscular dystrophy type 1 (DM1). Z-basivarsen is currently being evaluated in the Phase 1/2 ACHIEVE clinical trial in individuals with DM1.