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.

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...

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...

CMT Research Foundation Funds Cutting Edge Research to Deliver Therapeutics to the Peripheral Nervous System

Nov 19, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

The CMT Research Foundation has awarded nearly $100,000 to support a 13-month project to test a new therapeutic delivery system that may be capable of crossing the blood-nerve barrier and releasing therapeutic payloads to the Schwann cells and axons of the peripheral...

Researchers at Cyprus Institute of Neurology & Genetics Complete Third Milestone of CMT1A Gene Therapy Project

Aug 30, 2021 | CMT Research Updates, CMTRF Funded Research, Research News

The CMT Research Foundation is pleased to announce that Dr. Kleopas A. Kleopa and his team at the Cyprus Institute of Neurology & Genetics, Nicosia, have completed the third milestone of their 24-month CMT1A gene therapy project. CMT1A is caused by duplication of...

DTx Pharma, Inc. Completes CMT Research Foundation-Funded Project

Aug 27, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development

The CMT Research Foundation is pleased to announce that DTx Pharma, Inc. (DTx) has successfully completed their CMT Research Foundation-funded RNA-based therapy project. With funding from the CMT Research Foundation, DTx designed and tested a library of novel...