Latest 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 screening panels and the work […]
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 they need. As such, we […]
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 the U.S. Food and Drug […]
Read More ›Community Spotlight: Nicole Stickane Advocates for Newborn Screening in Texas
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 […]
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 are delighted to share with […]
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 and children 2 months and […]
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