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, Gene Replacement for CMT2S
The first session of the CMT Research Foundation's Gene Therapies Demystified series gave attendees the building blocks: what gene therapy is, why CMT is a compelling target, and what the science suggests about its potential. Now, the series moves from the...
Her Last Genetic Medicine Earned FDA Approval; Now She’s Leading the Way on CMT
The Food and Drug Administration recently approved Otarmeni, a gene therapy developed by Regeneron. The program was spearheaded by Meghan Drummond, PhD, who now serves as the Vice President of Research and Drug Development at the CMT Research Foundation. ...
CMTRF CEO Welcomed to Prestigious Cohort of Non-profit Research Leaders
The CMT Research Foundation’s CEO Laura MacNeill will participate in the 2026–2027 cohort of The FasterCures LeadersLink Program, a program from the Milken Institute. The program aims to help promising nonprofit research leaders worldwide build high-performing...
WEBINAR: Gene Therapies Demystified, Understanding the Fundamentals for CMT
https://www.youtube.com/watch?v=hrNkyIHed8U 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. The CMTRF launched Gene...
Alan Jackson Raffle Offers Exclusive Prizes, Benefits CMT Research Foundation
You can win Alan Jackson’s Corvette or a trip to his sold-out final concert through this raffle, with fundraising proceeds benefitting CMT Research Foundation. Full list of prizes includes: Grand Prize Alan Jackson’s 2013 Chevrolet Corvette Z16 Grand Sport Convertible...
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...
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...
