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 spinal muscular atrophy (SMA). In addition to our current support programs, Cure SMA is thrilled to launch a brand-new program offered to all affected individuals with SMA to help […]
Cure SMA Launches New Medical Alert Bracelet Support Program Read More »
Dear Members of the SMA Community, In light of the COVID-19 pandemic and the burden it has placed on our healthcare system, Biogen is working with health systems, institutions, physicians, patients and their families to navigate potential SPINRAZA® (nusinersen) dosing delays related to COVID-19. The health and safety of patients is our primary concern, and
Despite the new reality of quarantines, remote working, and social distancing, Cure SMA would like you to know that our work for the SMA community continues. Cure SMA remains vigilant in monitoring and responding to the needs of the SMA community, as your health, safety, and well-being is our top priority. We are launching a
Cure SMA Launches New COVID-19 Support Package Read More »
Cure SMA realizes that quarantining is essential for individuals and families who live with SMA, many of whom are experiencing hardships due to the COVID-19 pandemic. In response, we are pleased to launch a new program designed to help alleviate some of the financial burden for those in the SMA community who are affected in
COVID-19 Assistance Program Available to SMA Community Read More »
This article is the last in a series of Cure SMA grant announcements that have been shared throughout the past months. Cure SMA has awarded a $190,000 research grant to Stephen Meriney, Ph.D., at the University of Pittsburgh for his project titled, “A novel treatment targeting persistent neuromuscular dysfunction in a mild mouse model of
Dear SMA Community, As part of our ongoing partnership and following your request to receive important information about the risdiplam clinical development program, we wanted to update you on the status of regulatory filings and reviews of risdiplam by the U.S. Food and Drug Administration. In November 2019, the U.S. Food
Community Statement from Genentech on Risdiplam Approval Timeline Read More »
Biogen today announced that the first patient has been treated in the global clinical study, DEVOTE. The study is designed to evaluate the safety, tolerability, and potential for even greater efficacy of Spinraza (nusinersen) when administered at a higher dose than currently approved for the treatment of spinal muscular atrophy (SMA). The Phase 2/3 randomized,
AveXis, a Novartis company, announced a one-time infusion of Zolgensma® (onasemnogene abeparvovec-xioi) showed rapid, significant, and clinically meaningful therapeutic benefit in patients with spinal muscular atrophy (SMA) across a range of studies, including in patients treated pre-symptomatically, and sustained durability in patients now up to 5 years post-dosing and some patients more than 5 years
Dana Perella is a 9-year-old on a mission to raise $50,000 for spinal muscular atrophy (SMA) research in 2020. She began baking cookies to raise money for her friend Mila, who is affected by a rare and fatal genetic disorder called Batten. A year later, on behalf of her friend Ollie, she focused her cookie
Community Spotlight: Raising Money for Cure SMA, One Cookie at a Time Read More »
Cure SMA is pleased to announce the release of “Spotlight on SMA: The Spinal Muscular Atrophy Treatment Landscape,” the final supplement in a two-part series developed in collaboration with Neurology Reviews. The goal of this partnership is to increase awareness of SMA amongst healthcare providers, including current clinical guidelines and the need for prompt diagnosis
Cure SMA Launches Part 2 of “Spotlight on SMA” in Partnership with Neurology Reviews Read More »
