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
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, and LMI070—treat the underlying genetics of SMA. Gene therapy aims to replace the mutated in the SMN1 gene that causes SMA, and the other three
Cure SMA Releases 2016 Updates from Drug Programs in Clinical Trials Read More »
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 to be knowledgeable about the burden of proof required to support an FDA-approved treatment, and how that approval process works. As we get closer to
Cure SMA Posts Keynote Presentations on the NDA Process Read More »
Over the past few months, Cure SMA has announced a total of just over $2 million in new research funding. Our model is designed to fund both a breadth and a depth of projects, in order to continuing building up both a breadth and depth of programs in the drug pipeline. This includes funding for:
Cure SMA Funds $2 Million in New Research in 2015-2016 Read More »
The 2016 Annual SMA Conference, now in its 28th year, is set to kick off on June 16 and continue into a weekend full of programs and events that bring families, researchers, and healthcare providers together. This year’s conference is on track to have 25% more attendees than the last time we were at Disneyland!
The 2016 Annual SMA Conference is Here! Read More »
Cure SMA has awarded a $304,000 drug discovery grant to Livio Pellizzoni, PhD, at Columbia University. The award is for the project, “Pharmacological Inhibition of p38αMAPK as a Candidate Therapeutic Approach for SMA.” About the Project Dr. Pellizzoni and his colleagues at Northwestern University have identified a novel cellular pathway, called p38αMAPK, that is altered
