September 2017

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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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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