A record-number of individuals with spinal muscular atrophy (SMA), a neuromuscular disease, travelled to Orlando to attend Cure SMA’s national conference from June 29 to July 2. Several adults with SMA and families of children with SMA reported damage to their wheelchairs and other air travel hiccups that impacted their conference experience. These latest travel […]
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 Research & Clinical Care Meeting hosted by Cure SMA this week in Orlando, FL. Interim Clinical Outcomes from RESPOND RESPOND is an ongoing two-year, phase 4 open-label study to evaluate
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 its sustained efficacy and safety profile in children with Type 1 spinal muscular atrophy (SMA). FIREFISH is a two-part study in babies aged 1-7 months at the time of enrollment.
Chicago, June 30, 2023 – Cure SMA receives a surprise gift in the amount of $500,181 from the Luke 18:1 Foundation, a 501(c)3 organization founded in Dallas, TX, at the 2023 SMA Annual Conference. The Luke 18:1 Foundation was created by Daniel and Nicole Stickane in honor of their son, Luke, who was diagnosed with
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 play a fundamental role, today announced new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes at 36 months of treatment with
• 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 »
