Genentech, a member of the Roche Group, today presented 1-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1-7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint with 29% of infants (12/41) sitting without support for 5 seconds by month […]
Navigating the recent global health crisis, Coronavirus (COVID-19), has reinforced for us the importance of understanding what the SMA community is thinking, feeling, and experiencing. This allows us to more effectively support those impacted by SMA, both with the everyday needs you have and during greater public health challenges. Without this knowledge, we cannot advocate
Annual Cure SMA Community Update Survey Ready for Your Input Read More »
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
Cure SMA has been closely monitoring news and reports of the Coronavirus (COVID-19) outbreak, allowing us to make decisions about local events and offer new resources that support the SMA community. The current status of the pandemic and associated governmental regulations, restrictions, and advisories, as well as the need to protect those with SMA who
Dear SMA Community, Like many organizations across the United States and around the world, AveXis is closely monitoring the coronavirus disease (COVID-19) and its impact on patients, the SMA community, healthcare providers, and our employees and partners. We know that life with a rare genetic disease can bring significant challenges,
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 »
We want you to know that Cure SMA continues to be here for the entire SMA community. We remain vigilant in monitoring and responding to your needs, as your health, safety, and well-being are always our top priority. The following FAQs are designed to help address continued concerns expressed by members of the SMA community
Answering Your Coronavirus (COVID-19) Medical Questions 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,
