Front Page News
Community Spotlight: Braedon’s Newborn Screening Diagnosis
September is Newborn Screening Awareness Month, a chance to talk about the progress we have made in getting spinal muscular atrophy (SMA) added to newborn […]
Read More ›Biogen Q3 2020 Community Statement: COVID-19 Updates and Latest Milestones
Dear Members of the SMA Community, We continue to work with the healthcare community to help people and families living with SMA get the support […]
Read More ›Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of SMA
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that […]
Read More ›Genentech Releases Evrysdi™ FDA Approval Letter to the SMA Community
Dear SMA Patient Advocacy Community, As part of our ongoing partnership and following your request to receive updates about the risdiplam clinical development program, we […]
Read More ›Genentech Receives FDA Approval of Evrysdi (risdiplam) for the Treatment of SMA
Genentech, a member of the Roche Group, today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Evrysdi (risdiplam) to treat adults […]
Read More ›Cure SMA Launches New Medical Alert Bracelet Support Program
Along with funding SMA research and care, Cure SMA provides thousands of individuals and families with vital support and resources that help people navigate life […]
Read More ›Cure SMA to Meet with FDA for Critical Path Innovation Meeting
We are excited to share with the SMA community that the U.S. Food and Drug Administration (FDA) has granted Cure SMA a Critical Path Innovation […]
Read More ›Biogen Plans to Initiate Phase 4 Study Evaluating Benefit of Spinraza in Patients Treated with Zolgensma
Biogen Inc., today announced, it plans to initiate a global Phase 4 clinical study, RESPOND, to examine the clinical benefit and assess the safety of SPINRAZA (nusinersen) […]
Read More ›Community Spotlight: Ben Lou’s Formula for Life
Ben Lou is a 17-year-old from San Diego who is fluent in both English and Chinese. He is a math whiz and fascinated by science. […]
Read More ›Thank You for a Wonderful 2020 Virtual SMA Conference!
Cure SMA would like to thank everyone who attended last week’s 2020 Virtual SMA Conference! It was an impactful week of online workshops, networking, and […]
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