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...
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...
New FDA Draft Guidance Creates Opportunities for Gene Therapy Development
On June 2, 2026, the FDA's Center for Biologics Evaluation and Research released a draft guidance titled Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing. Gene therapy is one of the most promising avenues for...
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...
Gene Therapies Demystified: Terms to Help You
The CMT Research Foundation is hosting a three-part webinar series through Summer 2026 called Gene Therapies Demystified. The first installment is Understanding the Fundamentals for CMT, with CMTRF VP of Research and Drug Development Dr. Meghan Drummond. We've built a...
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...
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...
