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.
WEBINAR: Gene Therapies Demystified, Understanding the Fundamentals for CMT
One of the most rapidly expanding frontiers in Charcot-Marie-Tooth research is also one of the most challenging to keep pace with. Now, the CMT Research Foundation is making it accessible to everyone. On May 7th at 7 PM EST, the CMTRF will launch Gene Therapies...
CMTRF Funds Project to Measure if CMT Treatment is Working
The CMT Research Foundation has funded a new study at Johns Hopkins University to find a blood-based biomarker that could measure whether future CMT treatments are working. The research, targeted at subtype CMT2C, is led by Dr. Jeremy Sullivan and Dr. Charlotte...
New CMT Drug Enters Human Trials in China
ReviR Therapeutics, a previous CMT Research Foundation grantee, announced in March 2026 that the first human has been dosed with RTX-117, an experimental drug for Charcot-Marie-Tooth disease that ReviR says targets multiple CMT subtypes. The drug was administered in a...
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...
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 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,”...
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...
Dr. Charles Abrams Discovers That Inosine Treatment Leads to Benefits in a CMT1X Animal Model
Dr. Charles Abrams, Professor of Neurology and Rehabilitation at the University of Illinois, Chicago, has discovered that inosine could potentially reduce nerve inflammation and offer moderate functional benefits in preclinical animal models of Charcot-Marie-Tooth...