Dedicated to Research Committed to a Cure
Investing in innovative research to find treatments and cures for Charcot-Marie-Tooth disease.
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Latest CMT News
New FDA Draft Guidance Creates Opportunities for Gene Therapy Development
On June 2, 2026, the FDA's Center for Biologics Evaluation and Research released a draft guidance titled Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing.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...
Global CMT Research Convention: Highlights to Expect in 2026
The Global CMT Research Convention brings together the full spectrum of the Charcot-Marie-Tooth ecosystem in one place: leading scientists, biotech and pharma companies, investors, regulatory agencies and patients, all focused on accelerating treatments for the most...
Nobody Ran a Single Step at this CMTRF Fundraiser Run
Nearly 200 people gathered at Forgotten Star Brewing in Fridley, Minnesota, for one of the most unique fundraising events around: the Zero K Run, benefiting the CMT Research Foundation. The concept was simple. Participants got the full race-day experience, complete...
Register for the 6th Annual Global CMT Research Convention
CMT affects 1 in 2,500 people in the United States and more than 3 million people worldwide.
There is currently no cure for CMT.
WHO WE ARE
Accelerating a cure through research funding.
The CMT Research Foundation is a patient-led non-profit 100% focused on delivering treatments and cures for Charcot-Marie-Tooth, which affects 3 million people worldwide.
Since 2018, our donors and scientists have put CMT front and center in the pharmaceutical, biotech and investor communities, making a cure for all types of CMT a not-too-distant reality.
We have funded 6 therapeutics that are in preclinical or active trials
Featured Content
CMTRF Case Studies
Novartis' EKD060 compound for CMT1A is going to clinical trials
Novartis acquired CMTRF funed partner DTx Pharma in 2023 for $1 billion. DTx Pharma created a platform that delivers therapeutics to cells in a safe way. We introduced DTx Pharma to CMT and funded their first CMT research with a grant of $128,000. The results from the testing were positive and Novarts is now taking it to clincial trials.
CMTRF underwrites Augustine Therapeutics’ HDAC6 inhibitor. Now, the Belgian Pharmaceutical company is in a Phase 1 clinical trial.
CMTRF helped Augustine Therapeutics underwrite the development of novel potent and subtype-selective small-molecule inhibitors of the cytosolic histone deacetylase 6 (HDAC6) enzyme to reverse and inhibit the pathophysiological changes associated with neuromuscular diseases. Now Augustine Therapeutics has completed their Series A financing round and raised $85 million, which will be used to advance their lead candidate, AGT-100216, through a Phase I clinical trial. In May 2025, Augustine announced the dosing of their first patient in a Phase 1 clinical trial.
Armatus Bio licenses ARM-101 from CMTRF funded-partners Dr. Kleopa (Cyprus Institute of Neurology and Genetics) and Dr. Harper (Nationwide Children’s Hospital in Ohio) for clinical trials.
Drs. Kleopa and Harper developed a therapy that safely delivers miRNA, a PMP22 silencer, to the peripheral nervous system. Upon successful completion of their CMTRF-funded project, Armatus Bio licensed ARM-101 for further testing and now the therapeutic treatment is preparing for clinical trials.
Featured Content
We bring you the latest discoveries, innovative research and inspiring stories.
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 Lever...
Global CMT Research Convention: Highlights to Expect in 2026
The Global CMT Research Convention brings together the full spectrum of the Charcot-Marie-Tooth ecosystem in one pla...
Nobody Ran a Single Step at this CMTRF Fundraiser Run
Nearly 200 people gathered at Forgotten Star Brewing in Fridley, Minnesota, for one of the most unique fundraising ev...
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 out...
WEBINAR: Gene Therapies Demystified, Gene Replacement for CMT2S
The first session of the CMT Research Foundation’s Gene Therapies Demystified series gave attendees the buildin...
Gene Therapies Demystified: Terms to Help You
The CMT Research Foundation is hosting a three-part webinar series through Summer 2026 called Gene Therapies Demystif...
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 bu...
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...
CMTRF CEO Welcomed to Prestigious Cohort of Non-profit Research Leaders
The CMT Research Foundation’s CEO Laura MacNeil will participate in the 2026–2027 cohort of The FasterCures...
WEBINAR: Gene Therapies Demystified, Understanding the Fundamentals for CMT
One of the most rapidly expanding frontiers in Charcot-Marie-Tooth research is also one of the most challenging to ke...