SMA Patient-Focused Drug Development Meeting with FDA Set for April 18, 2017

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Tuesday, April 18, 2017, has been announced as the date for the SMA Patient-Focused Drug Development Meeting with the FDA. At the Patient Focused Drug Development (PFDD) Meeting, individuals and families from throughout our community will have the opportunity to share their stories with the FDA on a variety of topics important to our community. […]

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Cure SMA Assists Hundreds of Families in 2016

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Each year, Cure SMA provides thousands of families with vital support and resources to help them live active, engaged and hopeful lives.  Our programs provide tangible support for families affected by SMA, easing the difficulties of an SMA diagnosis, and showing each family that they are not alone and that there is hope. Our family

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Cure SMA Funding Results in 16 New Research Publications

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An important goal of our research funding strategy is to share scientific findings with the broader scientific community. To help accomplish this goal, scientists who recieve Cure SMA funding often publish their findings in peer-reviewed journals. Published articles allow the experiments and results to be reviewed and vetted by other scientists, who may then use

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2016 Research Year-in-Review: An Historic Year for Our Community

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This has been an historic year for the SMA community. On December 23, the FDA announced its approval of Spinraza, the first-ever approved treatment for SMA.  In addition to the approval of Spinraza, in 2016 the SMA drug pipeline reached notable highs. There are now: 18 active programs 6 programs in clinical trials 14 pharmaceutical

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Biogen Resources for the SMA Community

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Biogen has provided the following update on resources they have made available for the SMA community: Biogen recently announced that the U.S. Food and Drug Administration has approved SPINRAZA™ (nusinersen) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. At Biogen, we understand that there are many challenges that families living

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Join Us for an Upcoming Webcast about SPINRAZA TM (nusinersen)

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Please join Cure SMA, MDA and representatives from Biogen to learn more about SPINRAZA™ (nusinersen). As you know, SPINRAZA recently received FDA approval in the U.S. for the treatment of Spinal Muscular Atrophy (SMA) in pediatric and adult patients. In order to receive an update on SPINRAZA, please join this webcast, which is intended only

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Biogen Provides Community Statement and Q&A on the Approval of Spinraza for SMA

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Biogen provided the following statement on the approval of Spinraza for SMA: Dear Members of the SMA Community, Today, the SMA community achieved a goal that would not have been possible without the support and dedication of the families, physicians and researchers who have passionately pursued a treatment for Spinal Muscular Atrophy (SMA). We are

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Ionis Pharmaceuticals Issues Community Statement on the Approval of Spinraza

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Ionis Pharmaceuticals has issued the following community statement on the approval of Spinraza for SMA: Dear Members of the SMA Community, Today is a historic day for the SMA community with the approval of SPINRAZA™ (nusinersen) by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA). This is a

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FDA Approves Spinraza for SMA

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On December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA. We are thrilled to see our community’s efforts culminate in the approval of Spinraza: not only the first-ever approved treatment for this disease, but also one that addresses the underlying

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Cure SMA Participates in a Special FDA Listening Session

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Last week, Cure SMA participated in a listening session with FDA Commissioner Robert M. Califf, MD, MACC. The FDA organized the listening session in order to gain a better understanding of the current goals, priorities, and challenges of our community. Representatives from Cure SMA presented to the Commissioner and a panel of more than 25

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