Latest News
AveXis Receives FDA Breakthrough Therapy Designation for AVXS-101 (Gene Therapy) for SMA
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 granted […]
Read More ›Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports
Dr. Rashmi Kothary and his team have recently published a paper, “Differential induction of muscle atrophy pathways in two mouse models of spinal muscular atrophy” in the journal Scientific Reports. […]
Read More ›Adelphi Values’ RO7350A Interview Study
A note from Cure SMA: We’re passing on information about this new interview study to the families in our community. It provides an opportunity to make your opinions known to […]
Read More ›Cure SMA Awards $50,000 Drug Discovery Grant to Barrington Burnett, PhD, at Uniformed Services University of the Health Sciences
Cure SMA has awarded a $50,000 drug discovery grant to Barrington Burnett, PhD, at Uniformed Services University of the Health Sciences. The award is for the project, “Slowing SMN degradation […]
Read More ›Cure SMA Releases 2016 Updates from Drug Programs in Clinical Trials
At the Annual SMA Conference, representatives from the six programs currently in clinical trials gave an update on their programs. Of these six programs, four— AVXS-101 (gene therapy), nusinersen, RG7800/RG7916, […]
Read More ›Cure SMA Posts Keynote Presentations on the NDA Process
The ultimate goal in drug development is to get treatments approved for as many people as possible, as quickly as possible. Because these issues are complex, we want our community […]
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