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 with spinal muscular atrophy (SMA). In addition to our current support programs, Cure SMA is thrilled to launch a brand-new program offered to all affected individuals with SMA to help […]
Cure SMA Launches New Medical Alert Bracelet Support Program Read More »
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 Meeting (CPIM). This means that key leaders at the FDA will have the opportunity to communicate with clinical investigators, members of academia, industry, scientific groups, and patients to improve efficiency
Cure SMA to Meet with FDA for Critical Path Innovation Meeting Read More »
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) in infants and children with spinal muscular atrophy (SMA) who still have unmet clinical needs following treatment with gene therapy Zolgensma (onasemnogene abeparvovec). People with SMA do not produce enough survival
July 2020 marks the 2-year anniversary of when the U.S. Secretary of Health and Human Services recommended that SMA be added to state newborn screening panels. This month, you’ll hear about newborn screening milestones, stories, and steps you can take to ensure all states screen newborns for SMA. Shannon’s story of her family’s experience with
Community Spotlight: Avery’s Diagnosis from Newborn Screening Read More »
