Dedicated to Research Committed to a Cure

Investing in innovative research to find treatments and cures for Charcot-Marie-Tooth disease.

Donate Now
Research We Fund
Understand CMT as a recently-diagnosed patient
Find therapies under development
Take action
News

Latest CMT News

Explore more news
ASGCT

NMD Pharma Recruiting CMT Type 2 Patients for Phase 2 Clinical Trial

Read More
ASGCT

Live Webinar: The Story and The Science Behind Augustine Therapeutics’ $85M Raise: April 3, 2025: 7-8 pm ET

Register Now

Dr. Jerry Mendell, a leading gene therapy pioneer, will speak at "OHio, What a Night" on April 10, in Columbus

Learn More

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.

Donate Now
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 approaching clinical trials

See full research pipeline
Featured Content

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.

Learn More

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.

Learn More

CMTRF underwrites Augustine Therapeutics’ HDAC6 inhibitor. Now, the Belgian Pharmaceutical company is moving to phase ½ 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 raised $18.5 Million in a Series A First Closing, with proceeds from the Series A being used to advance Augustine’s first clinical candidate AGT100216 into a Phase 1/2 clinical trial in 2025.

Learn More
Featured Content

We bring you the latest discoveries, innovative research and inspiring stories.