Front Page News
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 […]
Read More ›Adult Scholarships Offered for the 2018 Annual SMA Conference
Cure SMA is pleased to offer scholarships to the 2018 Annual SMA Conference for all adults with SMA, thanks to a generous grant from The […]
Read More ›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 […]
Read More ›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. […]
Read More ›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 […]
Read More ›7th Annual Hope on the Hill Congressional Dinner Achieves $1 Million Milestone
This year’s 7th Annual “Hope on the Hill” Congressional Dinner will be held on Wednesday, November 29, bringing together families, government, and industry to mark […]
Read More ›SMA Advocates Testify at Federal Newborn Screening Meeting
On Wednesday, November 8, advocates testified at the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) in support of spinal muscular atrophy newborn […]
Read More ›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 […]
Read More ›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 […]
Read More ›New Clinical Trial Data Published in New England Journal of Medicine
Two new papers of clinical trial data for spinal muscular atrophy were published today in the November issue of the New England Journal of Medicine […]
Read More ›