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 emerging as a leader at BIO International Convention

Jun 29, 2022 | CMT Research Updates

The CMT Research Foundation meets with more than 30 companies at BIO and is recognized as a leader in patient driven research.

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...

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....

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...

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...

Gene Therapy Project for CMT2E has Completed Milestone 1 Successfully

Feb 23, 2022 | CMT Research Updates, CMTRF Funded Research, Research News

University of Missouri features CMT in a video about the project and the role of precision medicine to treat CMT. With the recent opening of their new NextGen Precision Health Institute, University of Missouri is quickly rising as a leader in precision medicine....

AcuraStem Completes CMT Research Foundation-Funded Project, Finds Eleven Potential Therapeutics for CMT2A

Feb 11, 2022 | CMT Research Updates, CMTRF Funded Research, Research News

The CMT Research Foundation is pleased to announce that the research team at AcuraStem has completed their CMT Research Foundation-funded drug screening project and has found eleven potential therapeutics for CMT2A. AcuraStem developed a platform to screen compounds...

Teams at Cyprus Institute of Neurology & Genetics and Nationwide Children’s Hospital Complete CMT Research Foundation-Funded Project, Resulting Therapeutic Now Moving into Pharmaceutical Development

Dec 16, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development

Update, May 17, 2022: This work is now published in The Journal of Clinical Investigation. The CMT Research Foundation is pleased to announce that Dr. Kleopas Kleopa, a renowned neuromuscular disease expert, CMT scientist, and clinician, and his team from the Cyprus...