Front Page News
Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children
Novartis recently announced data that reinforce the transformational benefit of Zolgensma® (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA). […]
Read More ›Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, announced an oral […]
Read More ›New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA
Genentech, a member of the Roche Group, today announced new interim data from two studies of Evrysdi® (risdiplam)—JEWELFISH and RAINBOWFISH. Genentech leads the clinical development of […]
Read More ›Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference
Biogen Inc., today announced new research supporting the continued development of an investigational higher dose of SPINRAZA® (nusinersen) and additional data reinforcing the strength of […]
Read More ›Ready to Go! Platform Open for 2021 Virtual SMA Conference Events
Cure SMA is excited to share that the platform for the 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting is […]
Read More ›Cure SMA Publishes Manuscript on SMA Awareness, Screening, and Referral Patterns Amongst Pediatricians
In a May 2021 issue of BMC Pediatrics, Cure SMA published a manuscript, titled “Awareness screening and referral patterns among pediatricians in the United States […]
Read More ›Cure SMA Announces 2022 Annual SMA Conference
Cure SMA is excited to announce that we will reunite the SMA community in-person for the 2022 Annual SMA Conference in Anaheim, CA–Thursday, June 16 […]
Read More ›Community Spotlight: Doug McCullough
Doug McCullough was raised on a dairy farm in Pennsylvania and now resides in Central New Jersey. He is a self-described “fun-loving guy.” “I take […]
Read More ›Community Spotlight: Chan Family
Ten-year-old Brady Chan from North Carolina has big goals that stem from his passion for video games. He is part of an e-gaming team, where his favorite games include “Brawl Starts” and […]
Read More ›Cure SMA Launches New Teen & Adult Independence Assistance Package – Check It Out Today!
Along with funding SMA research and care, Cure SMA provides thousands of individuals with SMA and their families with vital support and resources that help […]
Read More ›