Latest News
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, […]
Read More ›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. […]
Read More ›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 […]
Read More ›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 […]
Read More ›Survey Alert: SMA Family Caregivers
Family members of people with SMA play an active role in managing the daily lives of their loved one – from physical tasks like feeding, bathing, dressing, and more, to […]
Read More ›Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of apitegromab in those living with […]
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