Along with funding SMA research and care, Cure SMA provides thousands of individuals and families with vital support and resources that help people navigate life with SMA. Travel should be accessible for everyone but we know that is not often the case. With this in mind, Cure SMA is thrilled to launch some new travel […]
Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in both individuals previously treated and treatment-naïve to nusinersen Investigational regimen also shows more rapid slowing of neurodegeneration, as measured by neurofilament Biogen plans to submit regulatory applications around the world
Today, Scholar Rock announced plans to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorisation application (MAA) in Q1 2025. Scholar Rock, a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today
This week, Cure SMA and advocates with SMA celebrated Disability Pride and the 34th Anniversary of the Americans with Disabilities Act (ADA) with a special reception at the White House. The event marked a significant occasion to reflect on the progress made and the challenges that remain in achieving full equality and inclusion for people
Celebrating Disability Pride and the 34th Anniversary of the ADA at the White House Read More »
The positive study demonstrates the potential for investigational higher dose nusinersen regimen to advance the treatment of SMA. Biogen plans to submit for regulatory approval of this investigational dose regimen. Biogen today announced positive, topline data from the pivotal cohort (Part B) of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of a
During this SMA Awareness Month, Cure SMA recognizes all SMA community advocates – individuals with spinal muscular atrophy (SMA) and their families – who are raising awareness about SMA and advocating for SMA community priorities throughout the year. Cure SMA advocates play a crucial role in shaping policy and raising awareness. Cure SMA spoke with
Community Spotlight: Viola Dwyer, SMA Community Advocate from Pennsylvania Read More »
Cure SMA is pleased to announce the involvement of scientific leadership, in the 4th Scientific International Congress on SMA (March 14 – 16, 2024), the 2024 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conference (May 5 – 8, 2024), and the Annual SMA Research & Clinical Care Meeting (June 5 – 7, 2024). Our
Earlier this summer, at the Annual SMA Research and Clinical Care Meeting, Cure SMA highlighted six new basic research grants totalling $750,000. Cure SMA is now proud to announce another $750,000 will be awarded for additional research. With more than $85 million in funding to date in support of basic research grants and drug discovery
Whether you have SMA, have a loved one with SMA, are a researcher, care provider, industry partner, or are new to the SMA community, SMA Awareness Month is a time to come together to celebrate, honor, reflect, remember, and learn. The theme of SMA Awareness Month 2024 is ‘Breakthroughs Begin with You.’ It’s an opportunity for you
August is Spinal Muscular Atrophy (SMA) Awareness Month! Read More »
Cure SMA is pleased to announce the publication of “Newborn Screening and Birth Prevalence for Spinal Muscular Atrophy in the US” in JAMA Pediatrics on July 15, 2024. This paper reports the largest data collection of infants with SMA identified by newborn screening in the U.S. The objectives were: 1) to update the estimated birth
Cure SMA Publishes Update on the Birth Prevalence of Spinal Muscular Atrophy (SMA) Read More »
