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.

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...

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...

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...

Boost for CMT Research with a $3 Million MRFF Grant Awarded for Improving the Diagnosis of Rare Neurogenetic Diseases

Jul 18, 2021 | CMT Research Updates, News, Research News

Professor Marina Kennerson who directs the team at the Northcott Neuroscience Laboratory, ANZAC Research Institute in Sydney Australia is one of the lead researchers awarded a $3 million grant from the Medical Research Future Fund (MRFF) Genomics Health Futures...

CMT Research Foundation Targets CMT1X in Partnership with University of Illinois Chicago

Jul 8, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development, News, Research News

The CMT Research Foundation is partnering with the University of Illinois Chicago to test a potential therapeutic for CMT1X, the second most common form of CMT, behind only CMT1A in prevalence. CMT is one of the most common inherited neurological disorders, affecting...

CMT Research Foundation partners with Biotechnology Company AcuraStem and CMT Researcher Alessandra Bolino to test new drug in CMT4B1

Jun 27, 2021 | About CMT, CMT Research Updates, CMTRF Funded Research, Drug Development

The CMT Research Foundation is proud to announce our latest project, a partnership between AcuraStem, a patient-based drug discovery platform company, and Dr. Alessandra Bolino, a renowned expert in CMT and the head of the Human Inherited Neuropathies Unit at the...

CMT Research Foundation To Host First Global Research Convention

Jun 15, 2021 | CMT Research Updates, CMTRF Funded Research, Drug Development, News, Press Releases, Research News

Several Hundred Charcot-Marie-Tooth* World Leaders Gather to Accelerate Collaboration and Advancements in Research; Pharma Partner DTx to Review Significant Progress in Drug Delivery Gene Therapy Innovator and Pioneer Dr. James M. Wilson to Keynote ATLANTA (June...