Genentech, a member of the Roche Group, has shared new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 SMA. The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to […]
Biogen has shared new data from the Spinraza (nusinersen) clinical development program aimed at optimizing outcomes for people with SMA and advancing understanding of the disease. Exploring Opportunities to Optimize Treatment in SMA Building on the proven efficacy and well-established safety of Spinraza in a broad range of patients with SMA, the Phase 2/3 DEVOTE study is evaluating the
Last year presented the us with unique challenges, some we are still managing today. However, it has also reinforced the importance of understanding what the SMA community is thinking, feeling, and experiencing in their day-to-day. Knowing our community’s needs allows us to better serve you—whether it be advocating for comprehensive research, educating regulators, payers, and
Participate in Annual Cure SMA Community Update Survey – Open Now! Read More »
Cure SMA’s primary focus is supporting care of the highest value to individuals with SMA. As a result of treatments approved by the U.S. Food and Drug Administration (FDA), as well as successful initiatives like the Cure SMA Care Center Network and SMA Clinical Data Registry, we have made significant progress enhancing the lives of
Cure SMA Presents Data on Cure SMA Care Center Network and SMA Clinical Data Registry Read More »
Along with funding SMA research and care, Cure SMA is proud to provide thousands of individuals with SMA and their families with vital support they need to navigate life with SMA. In an effort to always be offering additional resources, we are thrilled to launch a new program for all individuals with SMA to help
Cure SMA Launches LifeVac Support Program to Help in Choking Event Read More »
Scholar Rock provided the below community statement on data for apitegromab (SRK-015) following the distribution of this company press release. Dear Members of the SMA Community, We are pleased to share 12-month topline results from our TOPAZ study, a Phase 2 clinical trial to evaluate the safety and efficacy of apitegromab (SRK-015) in individuals with
Scholar Rock Community Statement on TOPAZ 12-Month Topline Data Read More »
In honor of National Volunteer Month in April, Cure SMA is sharing stories and quotes from volunteers who support our programs and further grow our community. Cure SMA is incredibly grateful for the volunteers who keep this organization strong. You might know Nick Farrell as the newest Chairperson of the Cure SMA Board of Directors.
Community Spotlight: Nick Farrell Read More »
Dr. Chad Heatwole, a Cure SMA-funded researcher, and his team have published a paper in the Journal, Muscle Nerve, titled “The Spinal Muscular Atrophy Health Index (SMA-HI): A Novel Outcome for Measuring How a Patient Feels and Functions.” This project, funded by Cure SMA, focused on developing a multi-faceted, disease-specific, patient-reported outcome to measure an SMA
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 »
Scholar Rock Presents TOPAZ Interim Analysis Data for Apitegromab in SMA Scholar Rock recently shared 6-month interim analysis data from the TOPAZ Phase 2 clinical trial evaluating the potential of apitegromab, a highly selective inhibitor of latent myostatin, in improving motor function in patients with Types 2 and 3 spinal muscular atrophy (SMA). A this
Research Updates from Scholar Rock Read More »
