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 […]

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 Read More »

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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Congress Examines Ways to Improve Air Travel for People with Disabilities

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The air travel challenges of people with disabilities took center-stage this week in Congress through two separate congressional hearings. Cure SMA used the congressional action to once again highlight the experiences and recommendations of individuals with spinal muscular atrophy (SMA). This year, Congress must pass legislation related to the future of air travel in the

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Cure SMA Awards $75,000 Grant to Lyndsay Murray, PhD, University of Edinburgh

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Lyndsay Murray, PhD, was awarded $75,000 for her research project, “Evaluating the impact of sequential versus simultaneous administration of SMN-inducing compounds on motor unit recovery in mouse models of SMA.” Dr. Murray’s basic research grant is one of five awarded by Cure SMA in 2022 totaling $525,000. Meet Dr. Murray Tell us about yourself. I

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Biohaven’s Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy

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This week, Biohaven announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, for the treatment of spinal muscular atrophy (SMA). Fast Track designation enables important new drugs to reach patients earlier by facilitating more frequent communications with the FDA and expeditious review

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District of Columbia Starts Screening for SMA During Rare Disease Month

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Cure SMA is pleased to announce that the District of Columbia (DC) is now screening babies born in DC for spinal muscular atrophy (SMA). The DC Department of Health (DC Health) confirmed its screening news with Cure SMA. “DC Health is pleased to announce that as of February 1, babies born in the District of

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NEW REPORT: SMA Community Describes Air Travel Experiences as “Good, but mostly Bad, and Ugly”

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Despite federal laws and protections aimed at improving air travel for people with disabilities, a new report from Cure SMA highlights that the air travel experience for most individuals and families with spinal muscular atrophy (SMA), a neuromuscular disease, remains “mostly bad and ugly”. Individuals and families with SMA, a disease that significantly impacts physical

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Cure SMA Awards $150,000 Grant to Florence Rage, PhD, at the Institute of Molecular Genetics of Montpellier

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Florence Rage, PhD, was awarded $150,000 for her research project, “Deciphering the Mechanisms of RNA Transport along Axons to Understand the Progression of Spinal Muscular Atrophy (SMA).” Dr. Rage’s basic research grant is one of four awarded by Cure SMA in 2022 totaling $500,000. Meet Dr. Rage Tell us about yourself. I am a neuroscientist

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