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
CMTRF Launches the 2026 Global CMT Research Convention Virtual Experience
The CMT Research Foundation has launched a new way to watch the 2026 Global CMT Research Convention. The convention video platform puts the full program in one place, so you can revisit a session you attended or explore the convention for the first time. Browse by...
WEBINAR: A New Way to Fund CMT4C Research
The CMT Research Foundation is launching an innovative fundraising approach for CMT4C. Through the Community Leaders in 4C Program, patients, families, and supporters fund 4C research directly. 85% of your gift supports CMT4C research and community projects...
Virtual Workshop: Get the Most out of NODE
This workshop is built for scientists who are interested in using the Neuropathy Open Data Exchange (NODE), a resource from the CMT Research Foundation for peripheral nerve researchers to share data. Learn more about: Everything the platform can hold How to enter data...
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 funded 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.
CMTRF Launches the 2026 Global CMT Research Convention Virtual Experience
The CMT Research Foundation has launched a new way to watch the 2026 Global CMT Research Convention. The convention video platform puts the full program in one place, so you can revisit a session you…
Read More : CMTRF Launches the 2026 Global CMT Research Convention Virtual Experience
WEBINAR: A New Way to Fund CMT4C Research
The CMT Research Foundation is launching an innovative fundraising approach for CMT4C. Through the Community Leaders in 4C Program, patients, families, and supporters fund 4C research directly. 85% of your gift supports CMT4C research and…
Virtual Workshop: Get the Most out of NODE
This workshop is built for scientists who are interested in using the Neuropathy Open Data Exchange (NODE), a resource from the CMT Research Foundation for peripheral nerve researchers to share data. Learn more about: Everything…
Announcing NODE: A New Shared Home for Peripheral Nerve Research Data
The CMT Research Foundation (CMTRF) is excited to announce the launch of the Neuropathy Open Data Exchange (NODE), a new online tool built to help researchers share, compare, and make sense of data on peripheral…
Read More : Announcing NODE: A New Shared Home for Peripheral Nerve Research Data
WEBINAR: Introducing NODE, a New Shared Data Resource for Peripheral Neuropathy Research
In the era of AI, uncovering the secrets that lay buried beneath mountains of scientific data has never felt more possible. However, peripheral nerve research has long been slowed by valuable datasets sitting scattered across…
Read More : WEBINAR: Introducing NODE, a New Shared Data Resource for Peripheral Neuropathy Research
Global CMT Research Convention: Travel Guide
The 2026 Global CMT Research Convention Dates: September 24 to 26, 2026 Location: Cambridge, Massachusetts Host Hotel: The Royal Sonesta Boston, 40 Edwin H Land Blvd, Cambridge, MA 02142 Whether you're joining for the scientific…
New Paper Highlights CMTRF as a Model for Patient-Driven Research Organizations
A new reflection article published in FEBS Open Bio argues that patients and patient advocates belong at the bench, not just at the bedside. Laura MacNeill, CEO of the CMT Research Foundation, is among the…
Read More : New Paper Highlights CMTRF as a Model for Patient-Driven Research Organizations
What Makes This CMT Research Convention Our Most Hands-On Event Yet
The Global CMT Research Convention has always been a place where patients hear directly from the scientists and companies working on their behalf. This year, on Saturday, September 26 in Cambridge, MA, Patient Day…
Read More : What Makes This CMT Research Convention Our Most Hands-On Event Yet
Five Reasons to Consider a Planned Gift to CMTRF
Charcot-Marie-Tooth research is moving faster than ever, and planned gifts to the CMT Research Foundation (CMTRF) help make sure that momentum doesn't stop until there are treatments and cures for every person with CMT. A…
Read More : Five Reasons to Consider a Planned Gift to CMTRF
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.…
Read More : Project Report: A Negative Result Points to the Way Forward in CMT4B
