Research

Cure SMA Advocacy Campaign Leads to Congressional Support for More SMA Research

August 3, 2022
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Congress has recognized the unmet and new needs of individuals with SMA by including legislative language in must-pass legislation asking the National Institutes of Health […]

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Cure SMA to Hold Patient-Led Listening Session with FDA

August 1, 2022
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On Thursday, August 4, 2022, six members of the SMA community will have the opportunity to talk directly with the U.S. Food and Drug Administration […]

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Cure SMA Attends Spring 2022 Conferences to Promote Findings from Industry Collaboration Initiatives

June 9, 2022
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Cure SMA is pleased to announce the participation of scientific leadership in the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 13–16, 2022), […]

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FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy

May 31, 2022
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Genentech, a member of the Roche Group, today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to […]

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Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships

April 18, 2022
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Cure SMA is pleased to announce that we are accepting grant applications for funding of research projects and postdoctoral fellowships, under a competitive review by […]

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Cure SMA Announces Expanded Phase 7 of SMA Industry Collaboration

March 23, 2022
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Cure SMA is pleased to announce the launch of an expanded Phase 7 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted […]

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Biohaven Licenses a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA)

February 25, 2022
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Biohaven Pharmaceutical Holding Company Ltd., announced today that it entered into a worldwide license agreement with Bristol Myers Squibb for the development and commercialization rights to […]

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International Day of Women and Girls in Science

February 10, 2022
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Did you know? February 11 is International Day of Women and Girls in Science! While we’ve seen significant progress related to participation of women and […]

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Biogen Exercises Option with Ionis to Develop and Commercialize Investigational ASO for SMA

January 26, 2022
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      Biogen Inc. and Ionis Pharmaceuticals, Inc., recently announced that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license […]

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Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With SMA

January 25, 2022
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Genentech, a member of the Roche Group, recently announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new […]

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