Latest News
New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy
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 […]
Read More ›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
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 […]
Read More ›Luke 18:1 Foundation Donates $500,000 to Cure SMA to Support Groundbreaking SMA Research and Care
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 […]
Read More ›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
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 […]
Read More ›Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA
• 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 […]
Read More ›Community Update from Genentech
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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