News & Stories

See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.

CMT Research Foundation Funds Project with Alloy Therapeutics’ 82VS to Test Novel RNA Therapeutics for Charcot-Marie-Tooth Disease

Nov 13, 2024 | CMT Research Updates, CMTRF Funded Research

The CMT Research Foundation has invested over $500,000 in a program led by 82VS, the venture studio of Alloy Therapeutics, Inc. to discover and develop novel Antibody Oligonucleotide Conjugate (AOC) therapeutics for CMT. Oligonucleotide therapeutics offer promise for...

CMT Research Foundation Invests in Project at Nationwide Children’s Hospital to Develop Improved Gene Therapy Delivery Systems for Several Types of CMT

Aug 1, 2024 | CMTRF Funded Research

The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has invested in a research project to develop new vehicles for delivering gene therapies more efficiently into peripheral nerves. This...

Augustine Therapeutics Raises $18.5 Million in a Series A First Closing

Jun 26, 2024 | CMT Research Updates, CMTRF Funded Research

The Charcot-Marie-Tooth Research Foundation participated in the $18.5 million Series A funding round recently announced by Augustine Therapeutics. The financing was led by Asabys Partners, with the participation of Eli Lilly & Company, CMTRF, and current investors...

CMT Research Foundation Invests in ReviR Therapeutics Research to Pioneer Small Molecule Therapeutics for CMT1A

Jun 19, 2024 | CMT Research Updates, CMTRF Funded Research

The CMT Research Foundation has formed a strategic partnership with ReviR Therapeutics to advance small molecule therapeutics that modulate the expression of the gene that causes CMT1A. ReviR Therapeutics is developing a class of drugs called small molecule splice...

CMT Research Foundation Invests in Project to Test if a Commercially Available Drug is Effective Treatment for X-linked Charcot-Marie-Tooth Disease

Apr 23, 2024 | CMT Research Updates, CMTRF Funded Research, Press Releases, Research News

The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT), has invested in a project led by Dr. Charles Abrams, a Professor in the Department of Neurology and Rehabilitation at the...

CMT Research Update/NMD Pharma

Apr 1, 2024 | CMT Research Updates, Drug Development, Research News

NMD Pharma recently published data demonstrating that their drug candidate, NMD670, improves muscle function and neuromuscular transmission deficits in both animal models and patients. Dr. William David Arnold, a member of the CMTRF Scientific Advisory Board, who was...

Vanderbilt’s Charles Sanders Successfully Finds Molecules That Alter PMP22 Production or Cell Surface Trafficking; In Next Phase Will Test if They Can Improve CMT-like Problems in Schwann Cells

Mar 6, 2024 | CMT Research Updates, CMTRF Funded Research

CMT1A is caused by a gene-copying event that results in the overproduction of the peripheral myelin protein 22 (PMP22) in Schwann cells. The excess PMP22 protein fails to traffic normally to the cell surface and instead collects inside the cells as clumps of proteins...

CMT Research Foundation Partners to Advance Study of CMT1J by Dr. Stephan Zuchner

Feb 20, 2024 | CMT Research Updates, CMTRF Funded Research

The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT), has partnered with the 1J Foundation, a 501(c)(3) organization dedicated to finding a cure for patients with 1J, a newly identified...

Project Update: Nanite Looks to Advance Polymer-Based Encapsulation for CMT1A

Feb 9, 2024 | CMT Research Updates, CMTRF Funded Research

CMT 1A results from the duplication of the myelin protein 22 gene (PMP22) in Schwann cells. This causes excessive production of the PMP22 protein which disrupts the myelination process of peripheral nerves and ultimately results in axonal loss and muscle wasting over...

Updates on CMT Research

Dec 21, 2023 | CMT Research Updates, Research News

There have been a number of interesting developments in CMT research in the past few weeks. Here are three: Update 1: Pharnext recently announced topline results of its pivotal phase 3 (PREMIER trial) of PXT3003, its drug candidate for Charcot-Marie-Tooth 1A (CMT1A)...