New Clinical Trial Data Published in New England Journal of Medicine

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Two new papers of clinical trial data for spinal muscular atrophy were published today in the November issue of the New England Journal of Medicine (NEJM). NEJM is one of the most prestigious scientific journals in the US, and this dual publication reflects the scientific community’s increasing interest in and engagement with SMA research.  “Nusinersen […]

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Fall 2017 Issue of Compass Now Available

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The fall 2017 issue of Compass is now available online. This issue covers Phase II of the SMA Industry Collaboration, as well as highlights and results from the Patient Focused Drug Development Meeting with the FDA. Cure SMA Launches Phase II of the SMA Industry Collaboration The SMA Industry Collaboration is a multi-faceted partnership that

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Cure SMA Launches Benefit-Risk Survey to Address Important Issues in Treatment Access and Future Drug Approvals in SMA

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For the past several years, we have been working to bring your voices, experiences, challenges and hopes to inform regulatory authorities as key decisions are made on future therapies for SMA. Most recently, on April 18, 2017, the SMA community gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA to do just that.

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Cure SMA Announces Over $5 Million in New Research and Care Funding

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Cure SMA is pleased to announce $5 million in new research funding over the next 12 months. This funding will be used strategically to help accelerate research and ensure we are developing treatments for all types, ages and stages of SMA. This funding will also be used to increase patient access to treatments and improved

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Registration is Now Open for the 2018 Annual SMA Conference!

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Registration is now open for the 2018 Annual SMA Conference! This year’s conference will be held at the Hilton Anatole Hotel in Dallas, TX, from Thursday, June 14 through Sunday, June 17, 2018. We are excited to bring the conference to a central location and hope you can join us! Located in the Market Center

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Cure SMA Meets in Utah with State and Federal Elected Officials to Support Newborn Screening

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On Tuesday, Cure SMA staff members and family advocates, and clinicians from the University of Utah, met with Senator Orrin Hatch (R-UT) to discuss newborn screening for spinal muscular atrophy. Senator Hatch was an original sponsor of the Newborn Screening Saves Lives Act of 2007, which established federal programs to support states in the implementation

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Genentech/Roche Releases Clinical Trial Update for SUNFISH (RG7916)

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Genentech/Roche has provided the below community statement with clinical trial updates for SUNFISH (Type 2/3). The first patient is now enrolled in SUNFISH, as the trial advances to part two, which is designed to be a pivotal marketing registration trial.  Dear SMA Community, We are pleased to share an update on the SUNFISH study evaluating

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Recording of the September Webinar on Spinraza Updates Now Available

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A recording of last week’s webinar updating the community on Spinraza access and administration sites is now available online. A PDF of the webinar presentation is also available for download. An update on the current status of dosing in states across the US included the following information: As of September around 2,000 patients had been dosed, with

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AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process

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AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that based on review of data submitted, including the potency assay, it may initiate its planned pivotal trial of AVXS-101 for patients with

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Cure SMA Contributes Article to a Special Issue of the Journal Gene Therapy

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To celebrate the FDA approval of Spinraza, Cure SMA contributed an article to the September special issue of the journal Gene Therapy. The issue , “Spinraza and Advanced Therapies: a stakeholder special,” covers a variety of perspectives and includes comments and review papers from SMA research funders, people affected by SMA, experts in the advanced

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