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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Novartis Releases Update on LMI070 (Branaplam) Clinical Trial

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Novartis has released the following update for the LMI070 (branaplam) clinical trial, on behalf of the program team.  Dear SMA community, For years, our researchers have been working hard to develop a new treatment for SMA. One promising compound in our neuroscience pipeline is LMI070, which started development for the treatment of SMA a few

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Cure SMA Announces $5.05 Million in Research Funding for FY18

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Over the past fiscal year—from July 1, 2016, to June 30, 2017—Cure SMA has funded over $2.5 million in new research funding. This funding will be used strategically to help accelerate research, and ensure we are developing treatments for all types, ages, and stages of SMA. The areas funded include: Basic research, with a particular

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Applications for Cure SMA Medical Advisory Council Due October 15th

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Cure SMA is expanding its role in spinal muscular atrophy medical affairs at the national level through its Medical Advisory Council (MAC). The MAC sets the agenda for proactive, creative, and collaborative leadership on issues that improve the quality of medical care for those affected by SMA. The focus of the Council is to build

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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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