Latest News
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 in which protein growth factors […]
Read More ›Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA
• Alcyone’s ThecaFlex DRx™ System is an implantable medical device in development to enable routine subcutaneous administration of therapeutics to the cerebrospinal fluid• First stage of the pivotal study expected […]
Read More ›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, Summer is upon us and […]
Read More ›Congressional Committee Approves Legislation to Improve Air Travel for People with SMA and other Disabilities
This week, a key committee in the U.S. House of Representatives unanimously approved (63-0) aviation legislation that will make air travel more accessible for individuals with SMA and other disabilities. Accessible air […]
Read More ›Erin Trainor Memorial Fund will Match Donations for the funding of Care Centers
Double your donation to help double Cure SMA’s Care Center Network Care Centers guide our understanding of how SMA is changing, help us understand how care is being delivered, and […]
Read More ›Community Spotlight: Paula Lavigne and Chris Arnold
Thirteen years ago, Paula Lavigne and Chris Arnold’s adorable son, Wyatt, entered the world on July 9, 2010. While Paula and Chris could tell shortly after birth that Wyatt was […]
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