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 Announces FDA Orphan Drug Designation for NMD670
NMD Pharma has announced that they have been granted orphan drug designation by the Food and Drug Administration for NMD670, their novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of...
Maintaining Momentum: CMTRF’s Vision for 2025 and Beyond
Greetings and Happy New Year to you all. When I began my tenure as CEO of the CMT Research Foundation in October 2024, I was immediately inspired and impressed by the work of the Foundation. I was equally moved by the dedication of the greater CMT community,...
NMD Pharma Publishes Findings Supporting the Role of ClC-1 Inhibition in Charcot-Marie-Tooth Disease
NMD Pharma announced that they have published new clinical and preclinical data that provides evidence that targeting neuromuscular junction deficits could potentially improve muscle function in CMT patients. The peer-reviewed paper titled “Neuromuscular Transmission...
The Greatest Gift: Jillian Cabernel’s Story of Strength, Magic and the Hope for a Cure
The Greatest Gift: Jillian Cabernel, Vancouver, BC, Living With CMT December 2023 was a Christmas to remember for Jillian Cabernel. For the first time in her life, she truly felt the magic of the holidays. Jillian has spent her entire life living with...
Inaugural Zero-K Run Raises Awareness for CMT and CMTRF
When many people think of the word “race” they imagine a 5k, 10k and even a half-marathon. But, what about a new type of run that directly benefits patients with Charcot-Marie-Tooth disease and covers no distance at all—A Zero-K Run. A Zero-K Run is exactly what it...
Walking Manhattan to Find a Cure
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
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
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...
Updates on CMT Research
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
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...
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