News & Stories
See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.
CMT Takeaways from the MDA Conference
The Muscular Dystrophy Association Clinical & Scientific Conference in mid-March is one of the premier gatherings in the neuromuscular disease world, bringing together researchers, clinicians, industry, and advocates to share the latest science. Our research staff...
Using AI to Our Advantage: A Breakthrough Tool for Tracking CMT Progression
The CMT Research Foundation has invested in a research project with Dr. Wolfgang Pernice at Columbia University to develop a novel tool for clinical endpoint monitoring of Charcot-Marie-Tooth (CMT) disease progression entitled DANCER: Digital Assessment of Natural...
Update on NMD Pharma’s Phase 2a SYNAPSE-CMT Study
We are sharing an update from NMD Pharma regarding topline results from their Phase 2a clinical study called SYNAPSE-CMT. This clinical study explored whether the new, investigational drug ignaseclant (formerly NMD670), could provide benefit adults with...
William Skach, MD, Joins CMT Research Foundation Board of Directors
The CMT Research Foundation is pleased to announce that William Skach, MD, retired Executive Vice President and Chief Scientific Officer of the Cystic Fibrosis Foundation (CFF), has joined the Foundation’s Board of Directors. Dr. Skach is a clinically trained...
Lighting the Future: A Mother’s Fight for a Cure
The Greatest Gift: Kaileen Nelson, Canada, Living with CMT2D Every December, Kaileen looks for light. Not just the glittering bulbs strung across her Canadian neighborhood — but the kind of light that reminds her that hope still exists. Because for Kaileen, simple...
CMT Research Foundation to Benefit from a Portion of Ticket Sales from Alan Jackson’s Last Call: One More for the Road – The “Finale”
Alan Jackson is ready to serve up one more round before he retires from touring—and he’s inviting a few friends to join him! Last Call: One More for the Road - The Finale – the last full-length concert of Jackson’s touring career – will take place Saturday, June 27,...
CMT Research Foundation Funds Elpida Therapeutics to Launch Manufacturing of a Gene Therapy Drug for CMT4J
CMT Research Foundation announced they have funded Elpida Therapeutics to launch manufacturing of a gene therapy drug for Charcot-Marie-Tooth disease type 4J. The announcement came at the 2025 Global CMT Research Convention. “Our ELP-02 gene therapy is trial-ready,”...
Dr. Meghan Drummond Joins the CMT Research Foundation as Vice President of Research and Drug Development
Dr. Meghan Drummond, PhD, has joined the CMT Research Foundation as vice president of research and drug development. She will lead the Foundation’s research and drug development strategy, overseeing the advancement of promising therapeutic approaches for...
CMT Research Foundation Invests in the Next Generation of CMT Researchers
The CMT Research Foundation has awarded funding to four new research projects through its Emerging Researcher Award program, which provides small, high-impact grants to promising scientists in academia and industry. Spanning multiple Charcot-Marie-Tooth disease...
Applied Therapeutics to Meet With FDA on New Drug Application for the Treatment of CMT-SORD
Applied Therapeutics, who will be speaking at the 2025 Global CMT Research Convention, recently announced that they will be meeting with the U.S. Food and Drug Administration, during the third quarter of 2025, to discuss a potential New Drug Application for...