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Mar 21, 2026
Research from Germany and Australia shows zebrafish functional testing can identify false-positive SMA newborn screening results, preventing unnecessary treatment in rare cases where infants carry functional SMN1 gene variants.
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 19, 2026
Phase 1/2 MARINA trial results published in NEJM show del-desiran achieved approximately 40% reduction in toxic DMPK mRNA in muscle tissue and improvements in myotonia and functional measures in myotonic dystrophy type 1 patients, with acceptable safety profile.
Feb 25, 2025
Monthly patent reviews from April through December 2025 highlight notable drug discovery IP disclosures across targeted protein degradation, small molecule modulators, and emerging therapeutic targets in oncology, immunology, and neurology.
