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.

DTx Pharma

May 8, 2023 | About CMT, News

We are pleased share this story that published on May 3: https://patientworthy.com/2023/05/03/dtx-pharma-developing-cmt1a-rna-therapeutics/ One of the early programs CMTRF funded was DTx, a young company that had developed a new platform for RNA therapeutics. DTx was...

DTx Pharma Video – Using siRNA to Treat Charcot-Marie-Tooth Disease Type 1A

May 7, 2023 | CMTRF Funded Research, Drug Development, Research News

A Rare Disease Throughout Time

Feb 28, 2023 | About CMT, Life with CMT

More than 3 million people around the world have Charcot-Marie-Tooth (CMT) disease, but even with its quirky name, CMT is still relatively unknown. A progressive, degenerative disease involving the peripheral nerves that branch out from the brain and spinal cord to...

Dr. Arthur Suckow, DTx Pharma Inc. – FALCON

Nov 28, 2022 | CMTRF Funded Research, Drug Development

One of the first research teams the CMT Research Foundation (CMTRF) funded was DTx Pharma, Inc., and the project focused on delivery of genetic medicine to the myelinating Schwann cells of the peripheral nerves - a major scientific challenge to effectively treating...

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, 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...

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...

The Power of Scientific Collaboration – New Biomarkers for CMT

Mar 22, 2022 | About CMT, News, 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...