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.

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...

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....

Maximiliano Barrientos Joins CMT Research Foundation

Feb 15, 2022 | News, Press Releases

The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease* is pleased to announce that Maximiliano Barrientos has joined its Board of Directors. Mr. Barrientos is a Co-founder, Investor and CFO...

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...

Welcoming New Expert Advisors

Jan 25, 2022 | News

By Keith Fargo, Chief Scientific Officer The CMT Research Foundation’s Scientific Advisory Board is composed of distinguished scientists and clinicians who provide independent expert perspectives as key volunteers. Their contributions take several forms. Most visibly,...

CMT Research Foundation Elects Board Member Cleary Simpson to Chief Executive Officer

Jan 10, 2022 | News

By Patrick Livney, Board Chair and Co Founder As we move from being a startup to the leading foundation seeking to cure CMT, we can look back with great pride on our progress to date: We have raised almost $10 million in three years, funded 12 projects (with two in...