Dedicated to Research Committed to a Cure
Investing in innovative research to find treatments and cures for Charcot-Marie-Tooth disease.
September 25–27, 2025
Cambridge, MA
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News
Latest CMT News
Every Day Without Treatments Is a Day Too Long. Join Us To Fast-Track Research and Bring Hope to Families Living With CMT
CMT Research Foundation Invests in Asha Therapeutics to Test Novel Drug for CMT2A
Secure Your In-Person or Virtual Tickets to the 2025 Global CMT Research Convention Today
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 5 therapeutics that are in preclinical or active trials
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.
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.
Applied Therapeutics to Meet With FDA on New Drug Application for the Treatment of CMT-SORD
Applied Therapeutics, who will be speaking at the 2025 Global CMT Research Convention, recently announced that they w...
Dr. Charles Abrams Discovers That Inosine Treatment Leads to Benefits in a CMT1X Animal Model
Dr. Charles Abrams, Professor of Neurology and Rehabilitation at the University of Illinois, Chicago, has discovered ...
Dr. Yulia Grishchuk Joins CMTRF’s Scientific Advisory Board
CMT Research Foundation is pleased to announce that Dr. Yulia Grishchuk, Ph.D., has joined the Foundation’s Scienti...
CMT Research Foundation Invests in XtRNA Bio to Develop Gene Therapy for CMT2D
CMT Research Foundation has invested in a research project with XtRNA Bio aimed to develop a new viral gene therapy f...
A Father Who Defies ‘Impossible’: Joao Cardoso’s Journey with CMT
Joao Cardoso, Brittany, France, Living with CMT Joao Cardoso never imagined that he would be riding through the town ...
Siemens’ Ann Fairchild Joins CMT Research Foundation Board of Directors
CMT Research Foundation is pleased to announce that Ann Fairchild, executive vice president and general counsel of Si...
Applied Therapeutics Presents Findings From Phase 2/3 Clinical Trial of Govorestat in CMT-SORD
Applied Therapeutics recently presented findings from their INSPIRE Phase 2/3 clinical trial of govorestat (AT-007) f...
CMT Research Foundation Invests in Asha Therapeutics to Test Novel Drug for CMT2A
CMT Research Foundation has invested in a research project with Asha Therapeutics to investigate the therapeutic pote...
Vanderbilt University Researchers Identify Promising Compounds Targeting PMP22 Protein Expression, Paving the Way for New Treatments for CMT1A, CMT1E and HNPP
Researchers at Vanderbilt University have made significant progress in the development of small molecules aimed at tr...
Because of Luca: A Family’s Fight Against CMT
Tara, Luca, Liam and Lane, Louisiana, Living With CMT When Luca Haik was just four years old, his mother, Tara, sense...
