Research
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 […]
Read More ›Cure SMA to Fund Network of Prominent SMA Clinical Research and Treatment Centers
In the midst of the ongoing Coronavirus (COVID-19) global pandemic, Cure SMA is excited to announce its new funding for the Pediatric Neuromuscular Clinical Research […]
Read More ›Biogen Shares SPINRAZA Research Update
New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and Longer-Term Safety Across Broad Range of SMA Patients Biogen Inc. recently announced additional data from the SPINRAZA […]
Read More ›Biogen Releases Q2 Community Statement on Latest Milestones in Clinical Development Program
Dear Members of the SMA Community, In light of the COVID-19 pandemic and the burden it has placed on our healthcare system, Biogen is working […]
Read More ›Update from Scholar Rock on TOPAZ Phase 2 Clinical Trial of SRK-015 in Patients with SMA
Scholar Rock recently reported financial results for the first quarter ending March 31, 2020, highlighting progress and upcoming milestones for its pipeline programs—include the SRK-015 […]
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