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 research and ensure we are developing treatments for all types, ages, and stages of SMA. Funding Priorities As the SMA research landscape has developed and […]
Cure SMA Announces $2.5 Million in New Planned Research Funding Read More »
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 Breakthrough Therapy Designation for AVXS-101, the company’s lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 in pediatric patients. The Breakthrough
AveXis Receives FDA Breakthrough Therapy Designation for AVXS-101 (Gene Therapy) for SMA Read More »
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. This research was funded in part by grant from Cure SMA. Individuals with SMA do not produce survival motor neuron (SMN) protein at high enough
Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports Read More »
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 an influential group of decision makers, in keeping with our mission of supporting advocacy and awareness. Your participation in this is voluntary. In addition, Cure
Adelphi Values’ RO7350A Interview Study Read More »
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 to treat SMA.” Individuals with SMA don’t produce survival motor neuron (SMN) protein at high enough levels due to a mutation in the survival motor
