Front Page News
2017 Research Year-in-Review
Over our past fiscal year—from July 1, 2016, to June 30, 2017—Cure SMA has funded 28 projects with over $2.5 million in new research funding. […]
Cure SMA Advocates Continue to Advance Newborn Screening Efforts
SMA advocates across the country are working tirelessly with Cure SMA to educate state decision makers on newborn screening. In Maryland and Minnesota, the states’ […]
AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
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 […]
Adult Scholarships Offered for the 2018 Annual SMA Conference
Cure SMA is pleased to offer scholarships to the 2018 Annual SMA Conference for all adults with SMA, thanks to a generous grant from The […]
Cure SMA-Funded Research Results in 21 Published Journal Articles
An important goal of our research funding strategy is to share scientific findings with the broader scientific community. To help accomplish this goal, scientists who […]
Cytokinetics Publishes Clinical Trial Data for CK-2127107
Cytokinetics, Inc. recently announced the publication of results from three early clinical trials in healthy volunteers that evaluated safety, tolerability, pharmacokinetics, and pharmacodynamics of CK-2127107. […]
Cure SMA-Funded Researcher, Stephen Kolb, Publishes Paper
Cure SMA-funded researcher Stephen Kolb has published a paper in the journal Annals of Neurology, on the results of a study comparing infants affected by […]
7th Annual Hope on the Hill Congressional Dinner Achieves $1 Million Milestone
This year’s 7th Annual “Hope on the Hill” Congressional Dinner will be held on Wednesday, November 29, bringing together families, government, and industry to mark […]
SMA Advocates Testify at Federal Newborn Screening Meeting
On Wednesday, November 8, advocates testified at the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) in support of spinal muscular atrophy newborn […]
Biogen Issues Community Update on Spinraza
Biogen has provided the following community statement on Spinraza. Dear Members of the SMA community, As part of our commitment to the SMA community to […]