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.

Walking Manhattan to Find a Cure

Apr 24, 2024 | Life with CMT

From a young age, Rivka knew that there was something different about her compared to her 3 siblings. That difference was Charcot-Marie-Tooth disease. Meet Rivka, a young adult living with CMT1B in New York/Manhatten who is preparing to participate in this year's...

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...

Dr. Riann Egusquiza Joins The CMT Research Foundation as Director of Research

Apr 8, 2024 | About CMT, Press Releases

Riann Egusquiza (ryan egg-uh-ski-tha) PhD has joined The CMT Research Foundation as Director of Research, a newly created position. She will report to Cleary Simpson, CMTRF’s CEO, and be responsible for overseeing all CMTRF-funded research and scientific programming...

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...

Kayleena Speakman Joins The CMT Research Foundation as Communications Manager

Jan 4, 2024 | News, Press Releases

Kayleena Speakman has joined The CMT Research Foundation, a patient-led, non-profit focused on delivering treatments and cures for CMT as Communications Manager responsible for emails, blogs, social media posts and opinion pieces for the CMT community, donors,...

An Extraordinary Year and the Road Ahead

Jan 3, 2024 | News

As we begin 2024, I want to take a look back and celebrate the extraordinary year we had at the CMT Research Foundation. Since 2018, CMTRF has had an outsized impact on inspiring drug hunters to work on developing therapeutics for CMT and establishing itself as a key...

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)...