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.
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...
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. ...
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...
Applied Therapeutics to Meet With FDA on New Drug Application for the Treatment of CMT-SORD
Applied Therapeutics, who will be speaking at the 2025 Global CMT Research Convention, recently announced that they will be meeting with the U.S. Food and Drug Administration, during the third quarter of 2025, to discuss a potential New Drug Application for...
