Safety and efficacy of delpacibart zotadirsen in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the USA (EXPLORE44): a randomised, placebo-controlled, double-blind, phase 1/2 trial. (PubMed, Lancet Neurol) - Oct 15, 2026 - P1/2 | "Del-zota had an acceptable safety profile, produced increases in exon 44 skipping and dystrophin expression, and decreased biomarkers of muscle damage in participants with DMD44. Findings from this study support further investigation of del-zota in a phase 3 study to assess efficacy and long-term safety."
Clinical • First-in-human • Journal • P1/2 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy
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; Aravindhan Veerapandiyan; Brenda Wong; Carolina Tesi Rocha; Chamindra G Laverty; Craig M McDonald; Edward C Smith; Elizabeth J Ackermann; Han Phan; Jamie Eskuri; Josiah J Herzog; Kevin M Flanigan; Marc Morris; Philip Kovach; Steven G Hughes; Tara Carmack; Yiming Zhu; Yvonne Tami
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