Research
Biogen and Ionis Enter Into New Collaboration to Identify Novel Therapies for the Treatment of Spinal Muscular Atrophy
Biogen and Ionis Pharmaceuticals announced that they have entered into a new collaboration agreement to identify new antisense oligonucleotide drug candidates for the treatment of […]
2017 Research Year-in-Review
Over our past fiscal year—from July 1, 2016, to June 30, 2017—Cure SMA has funded 28 projects with over $2.5 million in new research funding. […]
AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
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 […]
Cure SMA-Funded Research Results in 21 Published Journal Articles
An important goal of our research funding strategy is to share scientific findings with the broader scientific community. To help accomplish this goal, scientists who […]
Cytokinetics Publishes Clinical Trial Data for CK-2127107
Cytokinetics, Inc. recently announced the publication of results from three early clinical trials in healthy volunteers that evaluated safety, tolerability, pharmacokinetics, and pharmacodynamics of CK-2127107. […]
Cure SMA-Funded Researcher, Stephen Kolb, Publishes Paper
Cure SMA-funded researcher Stephen Kolb has published a paper in the journal Annals of Neurology, on the results of a study comparing infants affected by […]
Biogen Issues Community Update on Spinraza
Biogen has provided the following community statement on Spinraza. Dear Members of the SMA community, As part of our commitment to the SMA community to […]
2017 World Muscle Society Congress Recap
From October 3rd through October 7th, Cure SMA attended the 22nd International Annual Congress of the World Muscle Society (WMS) in Saint Malo, France. WMS […]
Fall 2017 Issue of Compass Now Available
The fall 2017 issue of Compass is now available online. This issue covers Phase II of the SMA Industry Collaboration, as well as highlights and […]
Cure SMA Announces Over $5 Million in New Research and Care Funding
Cure SMA is pleased to announce $5 million in new research funding over the next 12 months. This funding will be used strategically to help […]