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
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’
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
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
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
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
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
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
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...
Boost for CMT Research with a $3 Million MRFF Grant Awarded for Improving the Diagnosis of Rare Neurogenetic Diseases
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
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...
Important Research News Announcements
- The Role of ESCRTs in Regulating Nervous System Function November 30, 2024
- Genomic Studies in Charcot-Marie-Tooth Disease July 31, 2024
- Charcot-Marie-tooth disease type 2A: An update on pathogenesis and therapeutic perspectives March 5, 2024
- Accelerate Clinical Trials in CMT (ACTCMT) Study March 1, 2024
- Creation of a Schwann Cell Gene Regulatory Network February 29, 2024
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