Author name: Leslie Humbel

Dr. Jacqueline Montes and colleagues at Columbia University—part of the Pediatric Neuromuscular Clinical Research Network (PNCRN) funded by Cure SMA—have published a paper in the journal Annals of Clinical and Translational Neurology, titled “Diminished muscle oxygen uptake and fatigue in spinal muscular atrophy.” This project, funded through the PNCRN, was an observational study evaluating muscle oxygen update […]

Cure SMA is pleased to announce its participation in the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference (March 15-18, 2021) and the American Academy of Neurology (AAN) 2021 Virtual Annual Meeting (April 17-22, 2021). Poster presentations at these meetings feature data from research initiatives launched by the Cure SMA Industry Collaboration (SMA-IC).

Last year presented the us with unique challenges, some we are still managing today. However, it has also reinforced the importance of understanding what the SMA community is thinking, feeling, and experiencing in their day-to-day. Knowing our community’s needs allows us to better serve you—whether it be advocating for comprehensive research, educating regulators, payers, and

Dr. Chad Heatwole, a Cure SMA-funded researcher, and his team have published a paper in the Journal, Muscle Nerve, titled “The Spinal Muscular Atrophy Health Index (SMA-HI): A Novel Outcome for Measuring How a Patient Feels and Functions.” This project, funded by Cure SMA, focused on developing a multi-faceted, disease-specific, patient-reported outcome to measure an SMA