Annual Cure SMA Community Update Survey Ready for Your Input

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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 […]

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COVID-19 Assistance Program Available to SMA Community

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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

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Grant Announcement: Cure SMA Awards $190,000 Grant to Stephen Meriney, Ph.D., University of Pittsburgh

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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

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Cure SMA Announces Cancellation of 2020 Annual SMA Conference and Research & Clinical Care Meeting

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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

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Community Statement from AveXis Regarding Commitment to SMA Community Amid COVID-19 Pandemic

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          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,

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Community Statement from Genentech on Risdiplam Approval Timeline

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        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

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Biogen Announces First Patient Treated With Higher Dose Of Spinraza In Phase 2/3 DEVOTE Study

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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,

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Zolgensma® Data Shows Rapid, Significant, Clinically Meaningful Benefit in SMA, Including Prolonged Event-free Survival, Motor Milestone Achievement and Durability, Now Up to 5 Years Post-dosing

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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

Zolgensma® Data Shows Rapid, Significant, Clinically Meaningful Benefit in SMA, Including Prolonged Event-free Survival, Motor Milestone Achievement and Durability, Now Up to 5 Years Post-dosing Read More »

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