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Gene Therapy Pioneer Jerry R. Mendell, M.D., Named to Inaugural TIME100 Health List of the 100 Most Influential People in Global Health

– TIME recognizes Dr. Mendell’s decades-long contributions in the treatment of neuromuscular disease and the advancement of gene therapy, including the first gene therapy for Duchenne muscular. ....

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Limb-Girdle Muscular Dystrophy Leaders Advance Collaborative Dialogue to Accelerate Drug Development to Address the Urgent Unmet Need in Limb-Girdle Muscular Dystrophy

/PRNewswire/ On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), convened a. ....

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Limb-Girdle Muscular Dystrophy Scientific Leaders - Together With the FDA - Will Come Together for a Drug Development Workshop on February 8, 2024

/PRNewswire/ On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), will be convening a. ....

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Gene transfer therapy safe, efficacious for children with Duchenne muscular dystrophy

Delandistrogene moxeparvovec improved motor function and was well-tolerated 4 years after treatment in a small cohort of children with Duchenne muscular dystrophy, according to a poster at the 2023 MDA Clinical & Scientific Conference.“Delandistrogene moxeparvovec is an investigational gene transfer therapy developed to address the root cause of Duchenne muscular dystrophy through ....

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Novartis shares Zolgensma long-term data demonstrating

Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusionAll. ....

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