Front Page News
Scholar Rock Issues Community Statement with Update on SRK-015 in Patients with Types 2 and 3 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 6-month […]
Read More ›Community Spotlight: Perspectives from Spencer Cook, Registered Accessibility Specialist
October is National Disability Employment Awareness Month, with an official theme of Increasing Access and Opportunity. Throughout the month, we have been posting stories about adults with […]
Read More ›Community Spotlight: Kayley Shade of Sioux Falls Storm
October is National Disability Employment Awareness Month, with an official theme of Increasing Access and Opportunity. Throughout the month, we will be posting stories about adults with […]
Read More ›NeurologyLive Article Now Available, Featuring an Interview with Cure SMA’s Dr. Mary Schroth
Through Cure SMA’s partnership with NeurologyLive, a new patient-centered article, “Early Intervention in SMA: How Access to Therapy Will Alter Disease Management As […]
Read More ›Cure SMA Publishes Paper in Partnership with NeurologyLive on Impact of COVID-19
Cure SMA has published a paper, titled “Spinal Muscular Atrophy and COVID-19: Guidance and Resources for Providers,” in partnership with NeurologyLive. The article […]
Read More ›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 […]
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