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.

Project Testing PIKfyve Inhibitor in CMT4B1 Clears First Hurdles

Jul 11, 2022 | About CMT, CMT Research Updates, CMTRF Funded Research, Research News

CMTRF is happy to report that a CMTRF-funded project designed to test a PIKfyve inhibitor in CMT4B1, a particularly devastating form of the disease, has passed the first stage and is moving on to the next stage, a preclinical trial in CMT4B1 model mice. Before the...

A New Trial for CMT – When Science Moves Quickly

May 18, 2022 | Drug Development, Research News

By Keith Fargo, Ph.D., Chief Scientific Officer, CMT Research Foundation The pace of science can seem excruciatingly slow. It can take many years, or even decades, for new ideas to result in clinical trials. Every so often, though, the stars align and a new...

Legacy – an Anthem for Patients with CMT

May 13, 2022 | Life with CMT

Recording-artist/songwriter/producer Dina Fanai AKA AVALONA, released Legacy today across all platforms Legacy was written as an anthem for patients with Charcot-Marie-Tooth disease (CMT), one of the world’s most common inherited neurological diseases. CMT causes...

2022 Global CMT Research Convention—Introducing the Scientific Program Committee

May 11, 2022 | CMTRF Funded Research

As we get ready for the 2022 Global CMT Research Convention, one of the most important events is the empanelment of a Scientific Program Committee, or SPC. What does the SPC do? In general, an SPC assists in the development of the scientific content for a convention....

CMT Mothers

May 4, 2022 | Life with CMT

The CMT Research Foundation knows that moms in the world of CMT have unique challenges and trials. And we salute their tenacity, patience, and unyielding dedication. Here are the stories of two CMT moms; one whose daughter lives with CMT and another who is a patient....

Shift Pharmaceuticals Completes CMT Research Foundation-Funded Project, Will Continue Development of Gene Therapy for CMT1A

May 4, 2022 | CMT Research Updates, CMTRF Funded Research, Research News

The CMT Research Foundation is pleased to announce that Shift Pharmaceuticals (Shift) has successfully completed their CMT Research Foundation-funded RNA-based therapy project. Shift created and tested a library of novel molecules designed to reduce the expression of...

Family Affected by CMT1A Makes a Cure Their ‘Endgame’

Apr 18, 2022 | About CMT, Life with CMT

Published by CMT News (slightly edited for clarity) It was an unexpected $1 million gift even for Kent and Michele Stahl. The Charcot-Marie-Tooth Research Foundation (CMTRF) was hosting a research convention in September when it announced Endgame, the organization’s...

Five Reasons Why We Think the End of CMT is in Sight

Apr 11, 2022 | About CMT, CMT Research Updates

Nearly every instance of CMT is caused by the mutation of a single gene and most genetic causes of CMT have been identified. The era of genetic medicine has already begun. The FDA first approved genetic medicines in 2016/2017. This set the foundation for a precision...

Living with CMT2E

Apr 6, 2022 | Life with CMT

My name is Lily Sander and I live with Charcot-Marie-Tooth type 2E. The road to my diagnosis wasn’t easy by any scale. I spent months as a small child in countless specialists' offices, anxiously waiting for an answer. At this time, I had severe clubfeet and could...

The Power of Scientific Collaboration – New Biomarkers for CMT

Mar 22, 2022 | Research News

At the CMT Research Foundation, we believe that more is achieved when people work together than when they work individually. This is especially true in biomedical research. Over the past few decades, a seismic shift has occurred in science with cooperation becoming...