CMT Research Foundation: 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
Project Report: A Negative Result Points to the Way Forward in CMT4B
A recent study found that a drug that helps other nerve diseases makes CMT4B1 worse, and figuring out why has opened a more promising path toward treatment, including a possible path forward for the related subtype CMT4B2. This work was funded jointly by the Muscular...
CMT Orgs Come together to Advocate for New Clinical Trial Framework
Organizations focused on Charcot-Marie-Tooth (CMT) disease have aligned with clinicians, and pharmaceutical companies to share a set of recommendations for how CMT clinical trials should be designed, with the recommendations published in Journal of the Peripheral...
The Promise Society: A New Way to Support CMT Research
There are moments when the future you've been fighting for finally comes into view. For the CMT Research Foundation, this is one of those moments. Because of our community, we are closer than ever to changing what it means to live with CMT. The research we're funding...
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.
Project Report: A Negative Result Points to the Way Forward in CMT4B
A recent study found that a drug that helps other nerve diseases makes CMT4B1 worse, and figuring out why has opened ...
CMT Orgs Come together to Advocate for New Clinical Trial Framework
Organizations focused on Charcot-Marie-Tooth (CMT) disease have aligned with clinicians, and pharmaceutical companies...
The Promise Society: A New Way to Support CMT Research
There are moments when the future you’ve been fighting for finally comes into view. For the CMT Research Foun...
Deadline Extended: Submit Your Abstract for the 2026 Global CMT Research Convention
We’re extending our abstract submission deadline for oral presentations. The original oral presentation abs...
Apply Now: Emerging Researcher Forum at the 2026 Global CMT Research Convention
The Emerging Researcher Forum is a closed, invite-only session held the morning of Day One of the 2026 Global CMT ...
The Full Agenda: 2026 Global CMT Research Convention
Charcot-Marie-Tooth disease drug development is at a pivotal moment. More active drug trials are underway than ever b...
WEBINAR: Behind the Scenes, 2026 Global CMT Research Convention Preview
Charcot-Marie-Tooth disease drug development is at a pivotal moment, with more active drug trials underway than ever ...
Taking Aim at CMT1J with a genetic medicine
The CMT Research Foundation, in partnership with the 1J Foundation, has funded a new study at the University of Mia...
Introducing the CMTRF Journal Club: A New Interactive Series for CMT Researchers
The CMT Research Foundation is launching a new Journal Club webinar series designed specifically for researchers wo...
Why You Should Be in the Room: Dr. Anna Greka to Keynote Global CMT Research Convention
Don’t miss Friday’s keynote at the Global CMT Research Convention (September 24 to 26, 2026, Cambridge...
