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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ASGCT

Every Day Without Treatments Is a Day Too Long. Join Us To Fast-Track Research and Bring Hope to Families Living With CMT

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ASGCT

CMT Research Foundation Invests in the Next Generation of CMT Researchers

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Secure Your In-Person or Virtual Tickets to the 2025 Global CMT Research Convention Today

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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.

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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 5 therapeutics that are in preclinical or active trials

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CMTRF Case Studies

We funded DTx Pharma (Novartis). Now their therapeutic treatment for CMT1A is going to clinical trials.

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. With our help, DTx was able to use successful results to get additional funding from NIH. Now, DTx Pharma was purchased by Novartis for $1 billion and the therapeutic treatment for CMT1A is going to clinical trials.

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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.

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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.

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