Clinical Trials
Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM
Genentech, a member of the Roche Group, announced in a press release that Evrysdi™ (risdiplam) data from the dose finding Part 1 of the pivotal […]
Read More ›Cure SMA Offer Perspectives on Combination Therapy for SMA
New developments in treatments for spinal muscular atrophy (SMA) over the last few years have changed what is possible for people with SMA, allowing them […]
Read More ›Biogen Provides SMA Community Update on DEVOTE Study
Below we have provided an update from Biogen regarding the status of its DEVOTE clinical trial. For more information about SPINRAZA (nusinersen), visit https://www.curesma.org/spinraza/. ++++++++++++++++++++ […]
Read More ›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 ›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 ›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 ›Biogen Plans to Initiate Phase 4 Study Evaluating Benefit of Spinraza in Patients Treated with Zolgensma
Biogen Inc., today announced, it plans to initiate a global Phase 4 clinical study, RESPOND, to examine the clinical benefit and assess the safety of SPINRAZA (nusinersen) […]
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 ›Cure SMA Publishes Article on SMA Clinical Trial Readiness Program in the U.S.
Cure SMA is proud to share with the SMA community that it has published a paper in the Orphanet Journal of Rare Diseases, […]
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 ›