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.
Introducing the CMTRF Journal Club: A New Interactive Series for CMT Researchers
The CMT Research Foundation is launching a new Journal Club webinar series designed specifically for researchers working in the CMT space. Each session will spotlight a recent, high-impact publication, with a short presentation from the research team followed by open...
FDA Grants Rare Pediatric Disease Designation to Investigational Therapy for CMT2S
On July 7, 2026, Vanda Pharmaceuticals announced that the FDA has granted Rare Pediatric Disease Designation to VCA-894A, an investigational antisense oligonucleotide (ASO) therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S). CMT2S is an inherited...
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...
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...
New FDA Draft Guidance Creates Opportunities for Gene Therapy Development
On June 2, 2026, the FDA's Center for Biologics Evaluation and Research released a draft guidance titled Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing.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...
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...
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...
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...
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. ...
