Originally published on April 1, 2014. REGENX Biosciences, LLC and AveXis, Inc. announce that they have entered into an exclusive agreement for the development and commercialization of products to treat Spinal Muscular Atrophy (SMA) using NAV rAAV9 vectors. Under the terms of the agreement, REGENX granted AveXis an exclusive, worldwide license, with rights to sublicense, […]

Originally published on April 24, 2014. Cure SMA invests in four research areas: Basic Research, Drug Discovery, Clinical Research, and Care Research. Cure SMA has invested $35 Million in research in the past decade alone. Right now we are providing funding for 24 different research projects. Later in 2014, new funding announcements will be made

Originally published on May 28, 2014. Cure SMA has been awarded a grant from The National Institute of Neurological Disorders and Disease (NINDS) and the Office of Rare Disease Research at The National Center of Advancing Translational Sciences (NCATS) of the National Institutes for Health (NIH) to support the 2014 SMA Research Group Meeting. We

Originally published on July 9, 2014. Cure SMA is pleased to announce that we are accepting grant applications for funding of research projects, under a competitive review by our Scientific Advisory Board.  Up to to $700,000 will be committed to new research projects in this round of funding, with other rounds of funding to be

Originally published on July 16, 2014. Cure Spinal Muscular Atrophy (SMA) is pleased to announce that we are accepting applications for 2014 funding of clinical care research projects, under a competitive review by our Medical Advisory Council (MAC). The goal of this program is to improve care and living with SMA. Program Overview: The deadline

Originally published on August 6, 2014. Christian Lorson PhD, investigator in the Bond Life Sciences Center and Professor of Veterinary Pathobiology at the University of Missouri, has been working on the development of a novel compound found to be efficacious in animal models of disease. The compound is an antisense oligonucleotide (ASO). It increases life

Originally published on August 20, 2014. Recently, we announced that Dr. Lyndsay Murray at The University of Edinburgh will receive a a Cure SMA basic research grant of $80,000 over two years, to explore the reasons causing motor neuron cell death in SMA. Motor neurons are nerve cells in the brain stem and spinal cord