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Novartis Presents New Zolgensma® Data Further Demonstrating Therapeutic Benefit
Novartis Gene Therapies today announced new interim data from the ongoing Phase 3 STR1VE-EU clinical trial for Zolgensma® (onasemnogene abeparvovec). The data demonstrates […]
Read More ›Genentech Presents New 2-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA
Genentech, a member of the Roche Group, today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi™ (risdiplam) in infants […]
Read More ›Community Spotlight: Xavier and Rose’s SMA Diagnosis
Carrie Menke is a South Dakota native who resides in Sioux Falls with her husband Tony and their four children — Xavier, Colette, Avila, and […]
Read More ›Novartis Issues Community Statement with Update on AVXS-101 Intrathecal Clinical Development Program
Dear SMA Community, We recently received important feedback from the U.S. Food and Drug Administration (FDA) following their review of data from the STRONG study […]
Read More ›Team Biogen Runs Over 22,000 Miles During SMA Awareness Month
Team Biogen hit the ground running throughout the month of August in honor of SMA Awareness Month. Together, the team logged […]
Read More ›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 […]
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