Latest News
Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA
Biogen and Ionis have been testing nusinersen in clinical trials and today announced that they will close ENDEAR, the Phase 3 trial testing nusinersen in infants with SMA type 1. […]
Read More ›Cure SMA Announces $2.5 Million in New Planned Research Funding
At the 2016 Annual SMA Conference, Cure SMA announced $2.5 million in new planned research funding over the next 12 months. This funding will be used strategically to help accelerate […]
Read More ›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 […]
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