SMA Community Update from Novartis Gene Therapies
We are pleased to share a community update on Novartis Gene Therapies’ Clinical Trial Program.
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FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy
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 include babies under two months old with SMA. The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of
Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships
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 our Scientific Advisory Board (SAB). Please click here to view the SAB roster. As we are now able to return to in-person activities for our SMA community, including conferences and
Cure SMA Announces Expanded Phase 7 of SMA Industry Collaboration
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 partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations, to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration,
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Cure SMA Support Services Now Expanded to Individuals and Families in Puerto Rico
Along with funding SMA research and care, Cure SMA provides thousands of individuals and families with vital support and resources that help people navigate life with SMA. In an effort to expand our current support services, we are thrilled to now offer all support packages and programs to the SMA Community living in Puerto Rico.
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Biohaven Licenses a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA)
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 taldefgrobep alfa, a novel, Phase 3-ready anti-myostatin adnectin. Taldefgrobep is the third development asset licensed to Biohaven from Bristol Myers Squibb and a clinical trial for SMA is expected to
International Day of Women and Girls in Science
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 girls in science, technology, engineering, and math (STEM), they are still underrepresented in these fields. For continued innovation and development in STEM, it’s imperative that women and girls feel empowered
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Biogen Exercises Option with Ionis to Develop and Commercialize Investigational ASO for SMA
Biogen Inc. and Ionis Pharmaceuticals, Inc., recently announced that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license to develop and commercialize BIIB115/ION306. The companies have a broad strategic collaboration to develop novel therapies to treat neurological disorders. “Combining Biogen’s expertise in neurology with Ionis’ leadership in antisense
Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With SMA
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 drug application (sNDA) for the use of Evrysdi® (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA). The sNDA submission incorporates interim data from the
Cure SMA Releases Updated Drug Pipeline
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 20 active programs, including three approved therapies. 10 pharmaceutical partners. 12 programs in clinical trials. An ever-increasing breadth of potential treatment approaches to SMA. Effective Treatments for All Ages, Stages and Types of SMA The updated version of this pipeline reflects
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