Industry Updates
Cure SMA Announces Expanded Phase 3 of Real World Evidence Collaboration
Cure SMA is pleased to announce the launch of an expanded Phase 3 of the Real World Evidence Collaboration. The collaboration was established to leverage […]
New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy
Biogen Inc. announced new SPINRAZA® (nusinersen) data aimed at answering critical questions for the spinal muscular atrophy (SMA) community. The data were presented at the SMA […]
Four-Year Follow-Up Data for Genentech’s Evrysdi Show Continued Increase in Number of Children With a Severe Form of Spinal Muscular Atrophy (SMA) Able to Sit, Stand and Walk
Genentech, a member of the Roche Group, announced today new long-term data for Evrysdi® (risdiplam) from the open-label extension (n=50) of the pivotal FIREFISH study, reinforcing […]
New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy
Below is an excerpt from a Scholar Rock press release: Scholar Rock, a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases […]
Community Update from Genentech
We are pleased to share the following SMA community letter from our partners at Genentech on their clinical development programs and initiatives. Dear SMA Community, […]
Novartis Gene Therapies Release May 2023 SMA Community Update
Novartis Gene Therapies recently provided an update on their clinical development program and initiatives. We invite you to check it out here!
Biogen Releases Q1 2023 Community Statement
Biogen recently issued a Q1 SMA Community Statement. Check it out here!
Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset
Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusion All children (100%) in […]
Biohaven’s Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy
This week, Biohaven announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a novel anti-myostatin adnectin, […]
Genentech Releases SMA Community Letter
Our industry partners at Genentech recently released an SMA community letter summarizing 2022 developments and initiates. Check it out here!