Genentech/Roche Releases Clinical Trial Update for RG7916

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Genentech/Roche recently provided the following community statement with clinical trial updates for SUNFISH (Type 2/3). The SUNFISH trial is advancing to part two, which is designed to be a pivotal, marketing registration trial. Dear SMA Community, We are focused on progressing our studies with our investigational molecule RG7916 with utmost speed and quality and are […]

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2017 SMA Awareness Month Recap

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The SMA community celebrated an impactful and advocacy-driven Awareness Month through a number of ways this August. Whether hosting candlelight vigils, meeting with state and local legislatures, holding fundraisers, coordinating MLB awareness games, and more, the community worked together to raise the general publics awareness of SMA. Advocacy Visits On August 2nd, members of the

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Cure SMA to Host Webinar Updating the Community on Spinraza Access

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On Tuesday, September 19, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST), Cure SMA will hold a webinar updating the community on the status of Spinraza access. Among the topics covered will be: The current status of dosing in states across the US  Administration sites, including site list information  Commercial and government insurance policies  Community resources

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2017 SMA Researcher Meeting Summary: Clinical Research Studies for SMA

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The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 attendees. The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research. The meeting also furthers research by building collaborations—including cross-disciplinary dialogue,

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2017 Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Disease Modifiers

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The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 attendees. The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research. The meeting also furthers research by building collaborations—including cross-disciplinary dialogue,

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Victor Dubowitz Honored at 2017 Researcher Meeting for Early Contributions to the SMA Field

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During the 2017 SMA Researcher Meeting, we honored one of our 2017 conference champions, Dr. Victor Dubowitz. Dr. Dubowitz’s early contributions to the SMA field were seminal and have been critical to our understanding of SMA pathology and clinical presentation. Dr. Dubowitz, originally from South Africa, is a neurologist, professor emeritus at the Imperial College,

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SMA Documentary Premieres at 2017 Rhode Island International Film Festival

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Life & Atrophy, a film about SMA, premiered at the Rhode Island International Film Festival on Saturday, August 12th. It was directed, produced, edited and photographed by Gareth Burghes. The film follows the McIntosh’s, whose son Miles has SMA Type II, as they navigate the diagnosis of SMA and as Miles continues to partake in

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Cure SMA-Funded Researchers, Diane Murrell and Deborah Boroughs, Publish Papers

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Two Cure SMA-funded projects have recently been published. Diane Murrell and her team published the paper, “The Experience of Families With Children With Spinal Muscular Atrophy Type I Across Health Care Systems” in the Journal of Child Neurology. Deborah Boroughs’ paper, “An Evaluation of a Continuing Education Program for Families of Ventilator-Dependent Children with Spinal

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2017 Researcher Meeting Summary: Regulation of Splicing and SMN Protein Expression

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The Annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 470 attendees. The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research. The meeting also furthers research by building collaborations—including cross-disciplinary dialogue,

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Centers for Medicare and Medicaid Services (CMS) Survey

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On August 3rd, as part of multiple days of advocacy visits, Cure SMA staff met with leaders from the Medicare division of the Centers for Medicare and Medicaid Services (CMS). The goal of the meeting was to advocate for robust reimbursement for Spinraza, as part of our ongoing coverage and payment policy project. While the

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