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.
Scientific Takeaways from the Peripheral Nerve Society’s Conference
The Peripheral Nerve Society hosted its annual conference in the Netherlands this June, and our research team went to learn about the latest advances, network with field leaders and accelerate progress for CMT treatments and cures. Here are our scientific team's...
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...
WEBINAR: Gene Therapies Demystified, Policy, Access, and the Road Ahead
https://youtu.be/AzOoaWPYpyc?si=44PoEHPxatjopWc- 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...
Project Report: A new way to deliver drugs to nerves in CMT
One of the biggest obstacles in CMT research is not a shortage of potential therapies, but a delivery problem: nerves are extremely difficult to reach through the bloodstream. Dr. Kelly Langert's team at Loyola University Chicago took a creative approach, asking...
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...
CMT1A Patients Dosed in Korean Phase 2a Drug Trial
ENCell, a South Korean company focused on cell and gene therapies, announced in May 2026 (as reported by Korean outlet The Bio News) that the first patient has been dosed in a Phase 2a clinical trial of EN001, a treatment candidate for Charcot-Marie-Tooth disease Type...
New Study at UC San Diego Aims to Identify Best Genetic Treatment for CMT2A
The CMT Research Foundation has funded a new study at University of California San Diego led by Dr. Uri Manor to build an end-to-end pipeline for finding and validating a genetic treatment for CMT2A. CMT2A is a progressive disease caused by mutations in a gene...
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...
