Front Page News
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 […]
Read More ›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 […]
Read More ›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 […]
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 […]
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 […]
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 […]
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, […]
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 […]
Read More ›Cure SMA Discusses FDA-Approved SMA Treatments & Dissects Combination Therapy
Cure SMA’s Jacqueline Glascock, Director of Research Programs, recently talked with Managed Healthcare Executive about information on the three FDA-approved SMA treatments and the need for […]
Read More ›Update on Genentech/Roche Initiation of MANATEE Clinical Study
We are pleased to share an update on Genentech’s, a member of the Roche Group, initiation of MANATEE, a new global Phase 2/3 clinical study […]
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