Front Page News
Cure SMA-funded Researcher Sara Custer Publishes Paper
Sara Custer, PhD, and her colleagues at Indiana University have published a new paper, “Altered mRNA Splicing in SMN-Depleted Motor Neuron-Like Cells” in the journal […]
SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA
Cure SMA is excited to announce that the SMA community has been granted a Patient Focused Drug Development Meeting with the FDA. At the Patient […]
Cure SMA and Ionis Announce 2016 Holiday Card Contest
Back by popular demand, Cure SMA and Ionis Pharmaceuticals are once again teaming up for a holiday card contest! This contest is open to children […]
AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 at the World Muscle Society Congress
AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an update on interim […]
Biogen Presents New Data at World Muscle Society Congress
New data from the clinical program for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), were presented by Biogen and Ionis Pharmaceuticals in the […]
Cure SMA Announces Newborn Screening Initiative in Partnership with MDA
Twice this year, Cure SMA has had the opportunity to testify before the federal Advisory Committee on Heritable Disorders on the need for newborn screening […]
Biogen and Ionis Release Community Statement on NDA Filing Completion
Biogen and Ionis Pharmaceuticals have provided the following community statement regarding the the completion of their NDA filing for nusinersen. Dear members of the SMA […]
Roche Launches New Phase 2 Clinical Trials
Roche has officially launched two Phase 2 clinical trials testing RO7034067 (also known as RG7916). The trials will begin recruitment in October. Earlier this month, […]
Catch Up on Research News from the 2016 Family Friendly Research Poster Session
Over the last decade, we’ve seen many advances in spinal muscular atrophy research, from new techniques in gene therapy to drugs that show promise in […]
Roche Provides New Update on Clinical Development of RG7916
Roche, PTC Therapeutics, and the SMA Foundation have released a community update on the development of their SMN2 splicing modifier clinical development program: Dear SMA […]