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.
Taking Aim at CMT1J with a genetic medicine
The CMT Research Foundation, in partnership with the 1J Foundation, has funded a new study at the University of Miami led by Dr. Stephan Züchner to evaluate a newly-identified genetic variant linked to Charcot-Marie-Tooth disease type 1J and to test whether a targeted...
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...
CMT Research Is Moving. Here’s What We’re Working On.
Charcot-Marie-Tooth disease progressively damages the peripheral nerves, causing muscle weakness, loss of sensation, and difficulty walking. There's no approved treatment. That's exactly why the CMT Research Foundation exists, and what we're working to change. ...
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New FDA Draft Guidance Creates Opportunities for Gene Therapy Development
On June 2, 2026, the FDA's Center for Biologics Evaluation and Research released a draft guidance titled Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing.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...
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...
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 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...
