Research
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 ›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 ›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 […]
Read More ›Genentech Announces 2-Year Risdiplam Data From SUNFISH and New Data From JEWELFISH in Infants, Children and Adults with SMA
Genentech, a member of the Roche Group, shared two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 […]
Read More ›Biogen Shares Results from Landmark NURTURE Study of Pre-Symptomatic SMA Patients Treated with Spinraza
Biogen, Inc., today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA), which is transforming […]
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