Latest News
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 Drug Administration (FDA) has notified […]
Read More ›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 Dhont Family Foundation. The scholarship […]
Read More ›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 receive Cure SMA funding often […]
Read More ›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. The data supports the ongoing […]
Read More ›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 SMA with healthy infants of […]
Read More ›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 the first-ever FDA approval of […]
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