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 Publishes Findings Supporting the Role of ClC-1 Inhibition in Charcot-Marie-Tooth Disease

Dec 18, 2024 | CMT Research Updates, Research News

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

Nov 18, 2024 | Life with CMT

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...

Actio Biosciences Receives Both Orphan Drug and Rare Pediatric Disease Designations From the FDA For Treatment of CMT2C

Aug 9, 2024 | CMT Research Updates, Drug Development

Actio Biosciences recently announced that they have received both orphan drug designation and rare pediatric disease designation for ABS-0871, a TRPV4 inhibitor, for the treatment of TRPV4+ Charcot-Marie-Tooth disease subtype 2C (CMT2C) from the U.S. Food and Drug...

Inaugural Zero-K Run Raises Awareness for CMT and CMTRF

Jul 19, 2024 | Life with CMT

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...

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...

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...