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.

NMD Pharma Receives FDA Clearance to Initiate Clinical Trial of NMD670 for CMT

Jun 18, 2024 | CMT Research Updates, Drug Development

NMD Pharma recently announced that it has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 2 clinical trial of NMD670 in patients living with CMT types 1 and 2. NMD670 is a...

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, 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)...

The Importance of Fundraising

Nov 14, 2023 | About CMT, Life with CMT

By: Miron Hall Because CMT is a rare disease, it often receives less attention and funding than more common medical conditions. Without adequate resources, researchers struggle to develop effective treatments and ultimately find a cure. This is where fundraising plays...

Update from the Novartis CMT1A Development Team

Oct 29, 2023 | About CMT, Drug Development

As you know, the CMT Research Foundation is proud to have introduced DTx leadership to Charcot-Marie-Tooth disease in 2019. We funded their first work in CMT to demonstrate the FALCON platform's potential to deliver a drug to the Schwann cells. And from there, the...