Author name: Zach Galati

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

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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 profile in children with Type 1 spinal muscular atrophy (SMA). FIREFISH is a two-part study in babies aged 1-7 months at the time of enrollment. […]

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 Read More »

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

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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 2023 SMA Annual Conference. The Luke 18:1 Foundation was created by Daniel and Nicole Stickane in honor of their son, Luke, who was diagnosed with

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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

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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 play a fundamental role, today announced new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes at 36 months of treatment with

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 Read More »

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

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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 to initiate in summer of 2023 for first 10 patients followed by enrollment of the remaining 80 patients in a second stage beginning in 2024

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

Congressional Committee Approves Legislation to Improve Air Travel for People with SMA and other Disabilities

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This week, a key committee in the U.S. House of Representatives unanimously approved (63-0) aviation legislation that will make air travel more accessible for individuals with SMA and other disabilities. Accessible air travel is a top priority for Cure SMA and the SMA community, which were recognized by the U.S. House committee for their advocacy and support of the legislation.  

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Erin Trainor Memorial Fund will Match Donations for the funding of Care Centers

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Double your donation to help double Cure SMA’s Care Center Network Care Centers guide our understanding of how SMA is changing, help us understand how care is being delivered, and help identify best practices in SMA treatment. We currently have nearly 30 Care Centers across the U.S., and with your gift and the generous support

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