Cure SMA-Funded Research Results in 21 Published Journal Articles

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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

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Cytokinetics Publishes Clinical Trial Data for CK-2127107

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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

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Cure SMA-Funded Researcher, Stephen Kolb, Publishes Paper

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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

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7th Annual Hope on the Hill Congressional Dinner Achieves $1 Million Milestone

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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

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SMA Advocates Testify at Federal Newborn Screening Meeting

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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

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Biogen Issues Community Update on Spinraza

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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

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2017 World Muscle Society Congress Recap

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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

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New Clinical Trial Data Published in New England Journal of Medicine

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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

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Fall 2017 Issue of Compass Now Available

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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 results from the Patient Focused Drug Development Meeting with the FDA. Cure SMA Launches Phase II of the SMA Industry Collaboration The SMA Industry Collaboration is a multi-faceted partnership that

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Cure SMA Launches Benefit-Risk Survey to Address Important Issues in Treatment Access and Future Drug Approvals in SMA

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For the past several years, we have been working to bring your voices, experiences, challenges and hopes to inform regulatory authorities as key decisions are made on future therapies for SMA. Most recently, on April 18, 2017, the SMA community gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA to do just that.

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