Latest News
Community Statement from AveXis Regarding Commitment to SMA Community Amid COVID-19 Pandemic
Dear SMA Community, Like many organizations across the United States and around the world, AveXis is closely monitoring the coronavirus disease (COVID-19) and its impact […]
Read More ›Community Statement from Genentech on Risdiplam Approval Timeline
Dear SMA Community, As part of our ongoing partnership and following your request to receive important information about the risdiplam clinical development program, we wanted to […]
Read More ›Answering Your Coronavirus (COVID-19) Medical Questions
We want you to know that Cure SMA continues to be here for the entire SMA community. We remain vigilant in monitoring and responding to your needs, as your health, […]
Read More ›Biogen Announces First Patient Treated With Higher Dose Of Spinraza In Phase 2/3 DEVOTE Study
Biogen today announced that the first patient has been treated in the global clinical study, DEVOTE. The study is designed to evaluate the safety, tolerability, and potential for even greater […]
Read More ›Zolgensma® Data Shows Rapid, Significant, Clinically Meaningful Benefit in SMA, Including Prolonged Event-free Survival, Motor Milestone Achievement and Durability, Now Up to 5 Years Post-dosing
AveXis, a Novartis company, announced a one-time infusion of Zolgensma® (onasemnogene abeparvovec-xioi) showed rapid, significant, and clinically meaningful therapeutic benefit in patients with spinal muscular atrophy (SMA) across a range […]
Read More ›Community Spotlight: Raising Money for Cure SMA, One Cookie at a Time
Dana Perella is a 9-year-old on a mission to raise $50,000 for spinal muscular atrophy (SMA) research in 2020. She began baking cookies to raise money for her friend Mila, […]
Read More ›