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.
New FDA Draft Guidance Creates Opportunities for Gene Therapy Development
On June 2, 2026, the FDA's Center for Biologics Evaluation and Research released a draft guidance titled Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing.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...
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...
CMT Takeaways from the MDA Conference
The Muscular Dystrophy Association Clinical & Scientific Conference in mid-March is one of the premier gatherings in the neuromuscular disease world, bringing together researchers, clinicians, industry, and advocates to share the latest science. Our research staff...
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...
Applied Therapeutics Presents Findings From Phase 2/3 Clinical Trial of Govorestat in CMT-SORD
Applied Therapeutics recently presented findings from their INSPIRE Phase 2/3 clinical trial of govorestat (AT-007) for the treatment of Sorbitol Dehydrogenase Deficiency, a subtype of Charcot-Marie-Tooth disease, at the Peripheral Nerve Society’s 2025 Annual Meeting...
Initial CMTRF Funding Leads Augustine Therapeutics to Raise $85 Million for CMT Clinical Trials
CMT Research Foundation funded partner Augustine Therapeutics recently announced that they completed their Series A financing round and raised $85 million, which will be used to advance their Charcot-Marie-Tooth disease drug candidate, AGT-100216, through a Phase I/II...
NMD Pharma Announces FDA Orphan Drug Designation for NMD670
NMD Pharma has announced that they have been granted orphan drug designation by the Food and Drug Administration for NMD670, their novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of...
NMD Pharma Publishes Findings Supporting the Role of ClC-1 Inhibition in Charcot-Marie-Tooth Disease
NMD Pharma announced that they have published new clinical and preclinical data that provides evidence that targeting neuromuscular junction deficits could potentially improve muscle function in CMT patients. The peer-reviewed paper titled “Neuromuscular Transmission...
