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.

Christopher Austin to Keynote CMT Research Foundation’s 2022 Global CMT Research Convention

Jul 12, 2022 | Press Releases

Christopher Austin, Founding Director of the National Center for Advancing Translational Sciences, to Keynote CMT Research Foundation’s 2022 Global CMT Research Convention ATLANTA (July 12, 2022) Christopher Austin, M.D., CEO of Vesalius Therapeutics, CEO-Partner of...

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...

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, 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, News

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...

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, News, Press Releases, 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 | 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...

CMTRF Research Efforts to Benefit from a Portion of Ticket Sales from Alan Jackson’s Last Call: One More for the Road Tour

Mar 11, 2022 | News, Press Releases

ATLANTA (March 11, 2022) CMT Research Foundation (CMTRF) the only nonprofit organization focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, will see its efforts boosted by country music superstar Alan Jackson's fans this year as...