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 notified the company that, based on review of data submitted, the company may initiate its planned Phase 1 clinical trial of AVXS-101 for patients with spinal […]
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 Dhont Family Foundation. The scholarship covers registration for one adult, age 18+, and one aide. Each year, we look forward to reuniting as a community at the Annual SMA Conference.
Adult Scholarships Offered for the 2018 Annual SMA Conference Read More »
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 receive Cure SMA funding often publish their findings in peer-reviewed journals. Published articles allow the experiments and results to be reviewed and vetted by other scientists, who may then use
Cure SMA-Funded Research Results in 21 Published Journal Articles Read More »
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. The data supports the ongoing Phase 2 trial of the drug in patients with spinal muscular atrophy (SMA). CK-2127107 is an investigational next-generation fast skeletal muscle troponin activator (FSTA) which
Cytokinetics Publishes Clinical Trial Data for CK-2127107 Read More »
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 SMA with healthy infants of the same age. This paper reported on the longitudinal results—that is, the change over time—between the two groups of infants. This paper follows an earlier
Cure SMA-Funded Researcher, Stephen Kolb, Publishes Paper Read More »
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 the first-ever FDA approval of a treatment for SMA, and look forward to the next stages for our community. Through this event, we hope to build momentum in the Washington
7th Annual Hope on the Hill Congressional Dinner Achieves $1 Million Milestone Read More »
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 screening efforts at the federal division of Health and Human Services. The advisory committee reviews conditions for addition to the Recommended Uniform Screening Panel (RUSP). The meeting included an update
SMA Advocates Testify at Federal Newborn Screening Meeting Read More »
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 provide ongoing and comprehensive communications about SPINRAZA® (nusinersen), we are pleased to share that the final results from ENDEAR, the Phase 3 study of SPINRAZA, were published in The New
Biogen Issues Community Update on Spinraza Read More »
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 is the premier annual congress on neuromuscular disorders, attended by established and young physicians, researchers, therapists and neuropathologists from all over the world. Cure SMA presented an overview of their
2017 World Muscle Society Congress Recap Read More »
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 (NEJM). NEJM is one of the most prestigious scientific journals in the US, and this dual publication reflects the scientific community’s increasing interest in and engagement with SMA research. “Nusinersen
New Clinical Trial Data Published in New England Journal of Medicine Read More »
