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,
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 »
As news about the Coronavirus (COVID-19) continues to evolve, Cure SMA has now decided to cancel all events (i.e., galas, dinners, Walk-n-Rolls, Summits of Strength) through the end of May. This decision took into account the most recent recommendation from the Centers for Disease Control and Prevention (CDC) to limit or cancel all gatherings of
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
