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Mar 15, 2026
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Mar 07, 2026
The FDA has denied or discouraged at least eight drug applications in the past year, including treatments for rare diseases, raising concerns about regulatory consistency and the future of experimental therapies.
Mar 06, 2026
Dyne Therapeutics is preparing a U.S. Accelerated Approval submission for z-rostudirsen in Duchenne muscular dystrophy and gearing up for a Phase 3 trial of z-basivarsen in myotonic dystrophy, while scaling operations to support potential commercialization.
Feb 17, 2026
Japan's Ministry of Health, Labour and Welfare granted Orphan Drug designation to Dyne Therapeutics' investigational treatment zeleciment-basivarsen for myotonic muscular dystrophy type 1, adding to existing designations in the U.S. and Europe.
