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...
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...
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...
Using AI to Our Advantage: A Breakthrough Tool for Tracking CMT Progression
The CMT Research Foundation has invested in a research project with Dr. Wolfgang Pernice at Columbia University to develop a novel tool for clinical endpoint monitoring of Charcot-Marie-Tooth (CMT) disease progression entitled DANCER: Digital Assessment of Natural...
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...
CMT Research Foundation Funds Elpida Therapeutics to Launch Manufacturing of a Gene Therapy Drug for CMT4J
CMT Research Foundation announced they have funded Elpida Therapeutics to launch manufacturing of a gene therapy drug for Charcot-Marie-Tooth disease type 4J. The announcement came at the 2025 Global CMT Research Convention. “Our ELP-02 gene therapy is trial-ready,”...
