This week, new data was presented at the World Muscle Society 2021 Virtual Congress. Check it out below! Scholar Rock presents two posters featuring additional analyses from the TOPAZ phase 2 trial of Apitegromab: Poster: Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (LBP.10) This […]
New Data Presented at World Muscle Society 2021 Virtual Congress Read More »
Biogen is committed to improving health outcomes for people impacted by spinal muscular atrophy (SMA) and generating data to assess if there are treatment pathways to address potential unmet needs. Today, we are excited to announce our intent to initiate a new global SMA clinical trial called ASCEND to understand whether an investigational higher dose
The advent of three approved SMN-upregulating therapeutics for spinal muscular atrophy has dramatically changed the natural history of the disease. There are, however, many uncertainties regarding timing of treatment initiation, response to treatment, and long-term outcomes. The identification of biomarkers, medical indicators that can inform us as to disease status, treatment response, and/or provide information
Cure SMA Convenes Working Group to Discuss Biomarkers for SMA Read More »
Cure SMA would like to thank everyone who attended the recent 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting. It was an impactful week of workshops, networking, and SMA community engagement. This year’s virtual conference week—the second virtual conference during these pandemic times—hosted nearly 2,500 registrants from over 65 countries. Cure SMA offered
Thank You for Another Fantastic Virtual SMA Conference! Read More »
When you first learned that you or your child had SMA, you probably began analyzing your home life, your or your child’s education, and other immediate concerns. In time, you realized that the needs would continue to evolve. How would you pay for medical expenses that insurance did not cover? What types of programs would
Financial Planning with a Disability Read More »
Biogen Inc., today announced new research supporting the continued development of an investigational higher dose of SPINRAZA® (nusinersen) and additional data reinforcing the strength of SPINRAZA’s clinical profile in improving the lives of individuals with spinal muscular atrophy (SMA) over the long term. “Intervention with SPINRAZA can meaningfully impact the trajectory of SMA, and we
Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference Read More »
Each year at the Annual SMA Conference, the session on Aquatic Therapy is among the most popular for attendees of all ages. Jennifer Martyn, PT, is one of the leaders of this educational and fun session. Normally this time of year, Jennifer and her staff would begin ordering new gear for the pool, labeling items
Understanding the Benefits of Aqua Therapy Read More »
Along with funding SMA research and care, Cure SMA provides thousands of individuals with SMA and their families with vital support and resources that help them navigate life with SMA. Following a successful 2018 launch of, and sustained interest in, our current Teen & Adult Support Package, Cure SMA is thrilled to offer a supplemental
Cure SMA Launches New Teen & Adult Independence Assistance Package – Check It Out Today! Read More »
Dr. Bakri Elsheikh and colleagues at The Ohio State University have published a paper in the Journal, Frontiers in Neurology, titled “Safety, Tolerability, and Effect of Nusinersen in Non-Ambulatory Adults with Spinal Muscular Atrophy.” This project, funded by Cure SMA, investigated the safety, tolerability, and treatment effect of nusinersen treatment in non-ambulatory adults with SMA. Non-ambulatory individuals
Biogen recently presented data about ongoing studies of SPINRAZA (nusinersen) in patients with Spinal Muscular Atrophy. Updates included the NURTURE, ENDEAR, SHINE, DEVOTE, and RESPOND clinical trials. Oral presentation details: Title: Predictive Factors of Nusinersen Treatment Response in Infantile-onset SMA: Results from the ENDEAR/SHINE Studies Presenter: Laurent Servais, MD, PhD Title: Nusinersen in Children with SMA Who
Nusinersen Research Updates Presented by Biogen Read More »
