• 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 to initiate in summer of 2023 for first 10 patients followed by enrollment of the remaining 80 patients in a second stage beginning in 2024 […]
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 so are a number of developments we are pleased to share with the SMA community. We are enjoying interacting with the community at various walks
Community Update from Genentech Read More »
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 travel is a top priority for Cure SMA and the SMA community, which were recognized by the U.S. House committee for their advocacy and support of the legislation.
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 help identify best practices in SMA treatment. We currently have nearly 30 Care Centers across the U.S., and with your gift and the generous support
Erin Trainor Memorial Fund will Match Donations for the funding of Care Centers Read More »
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 struggling, it took what felt like a long time in Wyatt’s short life for him to be diagnosed with SMA. Wyatt finally received his diagnosis
Community Spotlight: Paula Lavigne and Chris Arnold Read More »
Novartis Gene Therapies recently provided an update on their clinical development program and initiatives. We invite you to check it out here!
Novartis Gene Therapies Release May 2023 SMA Community Update Read More »
Cure SMA is excited to announce our newest publication geared toward adults with SMA. Thanks to contributions and feedback from adults with SMA in our community, we have collectively pulled together this booklet on adult-specific topics of importance. Check out the booklet here! Topics covered in Living Unlimited with SMA include: Planning for your Future
Living Unlimited with Spinal Muscular Atrophy: A Booklet for Adults with SMA Read More »
Thanks to the advocacy of individuals with spinal muscular atrophy (SMA) and others, the Centers for Medicare and Medicaid Services (CMS) now considers seat elevation systems in power wheelchairs as being “reasonable and necessary” for purposes of Medicare coverage. The federal agency updated its national policy to allow Medicare coverage for seat elevation systems in
Biogen recently issued a Q1 SMA Community Statement. Check it out here!
Biogen Releases Q1 2023 Community Statement Read More »
First patient treated in the ASCEND study evaluating the potential benefit of investigational higher dose nusinersen in children, teens and adults previously treated with Evrysdi® (risdiplam) Baseline characteristics indicate all nine infants and toddlers enrolled in RESPOND had suboptimal clinical status in ≥2 areas after receiving Zolgensma® (onasemnogene abeparvovec); there were no new safety findings with subsequent
Biogen Announces New Updates Across its SMA Research Program at 2023 MDA Conference Read More »
