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New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy

July 1, 2023
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Biogen Inc. announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community. The data were presented at the SMA Research & Clinical Care Meeting […]

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Four-Year Follow-Up Data for Genentech’s Evrysdi Show Continued Increase in Number of Children With a Severe Form of Spinal Muscular Atrophy (SMA) Able to Sit, Stand and Walk

July 1, 2023
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Genentech, a member of the Roche Group, announced today new long-term data for Evrysdi® (risdiplam) from the open-label extension (n=50) of the pivotal FIREFISH study, reinforcing its sustained efficacy and safety […]

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Luke 18:1 Foundation Donates $500,000 to Cure SMA to Support Groundbreaking SMA Research and Care

June 30, 2023
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Chicago, June 30, 2023 – Cure SMA receives a surprise gift in the amount of $500,181 from the Luke 18:1 Foundation, a 501(c)3 organization founded in Dallas, TX, at the […]

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New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy

June 30, 2023
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Below is an excerpt from a Scholar Rock press release: Scholar Rock, a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors […]

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Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA

June 28, 2023
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• Alcyone’s ThecaFlex DRx™ System is an implantable medical device in development to enable routine subcutaneous administration of therapeutics to the cerebrospinal fluid• First stage of the pivotal study expected […]

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Community Update from Genentech

June 22, 2023
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We are pleased to share the following SMA community letter from our partners at Genentech on their clinical development programs and initiatives. Dear SMA Community, Summer is upon us and […]

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