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 screening panels and the work still to be done. This month, you will hear about newborn screening milestones, stories, and steps you can take to ensure all states screen newborns […]
Community Spotlight: Braedon’s Newborn Screening Diagnosis Read More »
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 they need. As such, we wanted to share Biogen’s latest efforts on the following: We are committed to sharing information that will aid healthcare providers and individuals with SMA in
Biogen Q3 2020 Community Statement: COVID-19 Updates and Latest Milestones Read More »
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 the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA), a progressive, rare genetic disease that
In late July, the Texas Newborn Screening Advisory Committee held a meeting— virtually, of course —to hear an update on the status of statewide newborn screening for spinal muscular atrophy (SMA). State officials shared updates and members of the SMA community shared public comments. Nicole Stickane shared her family’s experience in support of adding SMA
Community Spotlight: Nicole Stickane Advocates for Newborn Screening in Texas Read More »
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 are delighted to share with you a much-anticipated milestone. Today, the U.S. Food and Drug Administration (FDA) has approved risdiplam for the treatment of spinal muscular atrophy (SMA) in adults
Genentech Releases Evrysdi™ FDA Approval Letter to the SMA Community Read More »
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 and children 2 months and older with spinal muscular atrophy (SMA). Evrysdi showed clinically meaningful improvements in motor function across two clinical trials in people with varying ages and levels
Genentech Receives FDA Approval of Evrysdi (risdiplam) for the Treatment of SMA Read More »
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 »
