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Individuals with SMA Describe New Travel Hiccups and Wheelchair Damage in Their Air Travels to Cure SMA National Conference
A record-number of individuals with spinal muscular atrophy (SMA), a neuromuscular disease, travelled to Orlando to attend Cure SMA’s national conference from June 29 to July 2. Several adults with […]
Read More ›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 […]
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