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Investing in innovative research to find treatments and cures for Charcot-Marie-Tooth disease.
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Latest CMT News
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, MA), featuring Dr. Anna Greka, founder of the Ladders to Cures (L2C) Scientific Accelerator at the Broad Institute of MIT and Harvard. Rare genetic diseases are...
FDA Grants Rare Pediatric Disease Designation to Investigational Therapy for CMT2S
On July 7, 2026, Vanda Pharmaceuticals announced that the FDA has granted Rare Pediatric Disease Designation to VCA-894A, an investigational antisense oligonucleotide (ASO) therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S). CMT2S is an inherited...
Submit Your Research: 2026 Global CMT Research Convention Accepting Abstracts
Mark your calendar: the 2026 Global CMT Research Convention will take place September 24 to 26 in Cambridge, Massachusetts, at the Royal Sonesta Boston. The event brings together patients, researchers, pharma companies, investors, and regulatory agencies working to...
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.
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...
FDA Grants Rare Pediatric Disease Designation to Investigational Therapy for CMT2S
On July 7, 2026, Vanda Pharmaceuticals announced that the FDA has granted Rare Pediatric Disease Designation to VCA-8...
Submit Your Research: 2026 Global CMT Research Convention Accepting Abstracts
Mark your calendar: the 2026 Global CMT Research Convention will take place September 24 to 26 in Cambridge, Massa...
Alan Jackson’s Final Tour Raises $2.25 Million for CMT Research
Alan Jackson’s “Last Call: One More for the Road” tour has officially wrapped, and the number is in...
CMT Research Is Moving. Here’s What We’re Working On.
Charcot-Marie-Tooth disease progressively damages the peripheral nerves, causing muscle weakness, loss of sensation, ...
Scientific Takeaways from the Peripheral Nerve Society’s Conference
The Peripheral Nerve Society hosted its annual conference in the Netherlands this June, and our research team went to...
One Last Round: Watching Alan Jackson Ride Off Into the Sunset
On June 27, Alan Jackson plays his final concert. After nearly four decades, 35 number one hits, and a catalog tha...
WEBINAR: Gene Therapies Demystified, Policy, Access, and the Road Ahead
The first session of the CMT Research Foundation’s Gene Therapies Demystified series gave attendees the building bl...
Project Report: A new way to deliver drugs to nerves in CMT
One of the biggest obstacles in CMT research is not a shortage of potential therapies, but a delivery problem: nerves...
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...
