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 »
Cure SMA has released the schedule for the 2020 Annual SMA Conference, which will be held at Disney’s Yacht & Beach Club Resorts in Orlando, Fla., from Thursday, June 11th thru Sunday, June 14th. As was communicated in last week’s Community Statement, at this time, we are hopeful that the coronavirus will be contained by this
2020 Annual SMA Conference Schedule is Released Read More »
This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA has awarded a $150,000 grant to Emily Welby, Ph.D., of the Medical College of Wisconsin for her project titled, ” The role of astrocytes in SMA motor neuron synapse defects.” Dr. Welby is a post-doctoral researcher working
This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA has awarded a $190,000 research grant to Umrao Monani, Ph.D., of Columbia University, for his project titled, “Genetic suppressors of the SMA phenotype.” Dr. Monani is associate professor of Neurology and Pathology & Cell Biology, and
This article is part of a series of Cure SMA grant announcements being shared throughout the winter/spring. Cure SMA and Cure SMA Canada have awarded a $100,000 research grant to Rashmi Kothary, Ph.D., at the Ottawa Hospital Research Institute, for his project titled, “Characterization of canonical disease features in a novel mouse model of SMA
