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At the 2026 MDA Conference, Atamyo Therapeutics reported encouraging nine-month data from the first patients treated in its Phase 1b/2 trial of ATA-200 for LGMD-2C/R5, a severe childhood-onset muscular dystrophy caused by γ-sarcoglycan deficiency. Muscle biopsies showed over 90% of fibers expressing the therapeutic protein, alongside sustained reductions in muscle-damage biomarkers and functional improvements. No serious adverse events were observed in the four patients treated to date.
Atamyo Therapeutics announced a new milestone in its Phase 1b/2 clinical trial of ATA-200, with a fourth patient now dosed in the study evaluating its gene therapy for γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5. The company recognized patient advocacy organization CureSCG, whose support — alongside the Dion Foundation — has helped advance the program for families affected by this rare and progressive neuromuscular disease.
