April 2023

Biogen Announces New Updates Across its SMA Research Program at 2023 MDA Conference

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First patient treated in the ASCEND study evaluating the potential benefit of investigational higher dose nusinersen in children, teens and adults previously treated with Evrysdi® (risdiplam) Baseline characteristics indicate all nine infants and toddlers enrolled in RESPOND had suboptimal clinical status in ≥2 areas after receiving Zolgensma® (onasemnogene abeparvovec); there were no new safety findings with subsequent […]

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Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset

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Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusion All children (100%) in the presymptomatic intravenous cohort of LT-002 maintained or achieved all assessed motor milestones, including independent walking To date, more than 3,000 children with spinal muscular atrophy have been treated with

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Cure SMA Awards $100,000 Grant to Saravanan Arumugam, PhD, Universidad de Seville

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Saravanan Arumugam, PhD, was awarded $100,000 for his research project, “Role of Cytosolic and Mitochondrial Ca2+ in the Pathogenesis of Spinal Muscular Atrophy.” Dr. Arumugam’s basic research grant is one of five awarded by Cure SMA in 2022 totaling $500,000. Meet Dr. Arumugam Tell us about yourself. I am a postdoctoral fellow in the Department

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Cure SMA Announces Expanded Phase 8 of SMA Industry Collaboration

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Cure SMA is pleased to announce the launch of an expanded Phase 8 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration, we fund

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