Registration Opens for the SMA Patient-Focused Drug Development Meeting with the FDA

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Registration is now open for the SMA Patient-Focused Drug Development (PFDD) meeting with the FDA. The PFDD meeting will be held at the College Park Marriott, near Washinton DC, on Tuesday, April 18.  There are two primary ways to participate in this meeting:  Attend in person. Registration for in-person attendance is free, but seating is […]

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Spinraza Access Update Webinar

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On Tuesday, February 14, at 1:00pm EST (10:00am PST/11:00am MST/12:00pm CST), Cure SMA hosted a webinar updating the community on the status of Spinraza access. Among the topics covered were adminstration sites, insurance coverage, and community resources and support programs.  We were happy to host an open, candid, and informal discussion of where our community

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Cure SMA Launches Community Survey to Address Urgent and Important Issues in SMA Treatment

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For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes and challenges. We know that the voice of our community is powerful. By sharing our stories, we can communicate our priorities to the FDA and regulators, provide insight into daily life with SMA and the ongoing

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Biogen Presents New ENDEAR Data Showing SPINRAZA (nusinersen) Significantly Reduces Risk of Death or Permanent Ventilation

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Biogen presented new data from the Phase 3 ENDEAR study of SPINRAZA™ (nusinersen), which demonstrated a statistically significant reduction in the risk of death or permanent ventilation in SPINRAZA-treated infants with spinal muscular atrophy (SMA), compared to untreated infants. The data was presented at the British Pediatric Neurology Association (BPNA) annual conference in Cambridge, UK,

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Updated SMA Drug Pipeline Released as First Commercial Patient Receives Spinraza

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With the news today that the first-ever patient has received the first commercial Spinraza dose, we are releasing an updated version of our SMA drug pipeline. This new version reflects the approval of Spinraza, as well as the true start of a key stage to ensure that more and more patients are able to gain

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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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