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.

CMT Research Foundation partners with Samsara Therapeutics to develop a novel class of drugs in CMT1A

Oct 13, 2022 | About CMT, CMT Research Updates, CMTRF Funded Research, Research News

The CMT Research Foundation is pleased to announce a new project with Samsara Therapeutics, a US/UK-based biotech company that is developing a novel class of drugs for CMT1A. CMT1A is caused by duplication of a stretch of DNA that includes the PMP22 gene, and people...

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...

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....

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...

Shayla Hammock, living with CMT1A

Feb 25, 2022 | Life with CMT

I inherited CMT1A through my family, although I was unaware of what was affecting me until I was seven years old. As a baby, I was unable to lift my head at the same time as my peers and it took me a little longer to learn to walk. In fact, it seemed like every stage...

Living with Muscle Weakness is Not Rare to Me

Feb 18, 2022 | Life with CMT

Krista Allison, living with CMT4J I was born with Charcot-Marie-Tooth disease (CMT) and my symptoms started to appear about 12-months-old when I began to learn to walk. Since the age of 15, I have had roughly 12 foot surgeries to correct foot abnormalities, although I...