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.
One Last Round: Watching Alan Jackson Ride Off Into the Sunset
On June 27, Alan Jackson plays his final concert. After nearly four decades, 35 number one hits, and a catalog that helped define what country music sounds like, he is stepping away from the stage on his own terms. Several years ago, Alan Jackson opened up about...
Global CMT Research Convention: Highlights to Expect in 2026
The Global CMT Research Convention brings together the full spectrum of the Charcot-Marie-Tooth ecosystem in one place: leading scientists, biotech and pharma companies, investors, regulatory agencies and patients, all focused on accelerating treatments for the most...
Nobody Ran a Single Step at this CMTRF Fundraiser Run
Nearly 200 people gathered at Forgotten Star Brewing in Fridley, Minnesota, for one of the most unique fundraising events around: the Zero K Run, benefiting the CMT Research Foundation. The concept was simple. Participants got the full race-day experience, complete...
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...
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...
