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Jul 23, 2026
Transcripta Bio raised $24M backed by Mayo Clinic and Omnimed for AI-driven neurological and neuromuscular therapies. The funds will support IND-enabling studies and clinical preparation for ASD and FSHD programs.
Mar 26, 2026
Researchers have identified KHDC1L as a circulating biomarker for DUX4 activity in facioscapulohumeral muscular dystrophy (FSHD). The discovery could enable non-invasive blood-based monitoring of disease progression instead of muscle biopsies. The finding emerged from collaboration between academic researchers and Avidity Biosciences scientists.
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 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.
