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.
Project Testing PIKfyve Inhibitor in CMT4B1 Clears First Hurdles
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...
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...
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...
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...
CMT Research Foundation To Host First Global Research Convention
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...
Promising Research Results Point to Potential Cures for Two Most Common Forms of CMT
Momentum is building in the development of gene therapies for Charcot-Marie-Tooth disease. Landmark back-to-back research publications offer innovative approaches to potentially curing the two most common forms of CMT. Here is what the findings showed and what they mean for CMT patients and families.
Important Research News Announcements
- 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
- Activation of XBP1s attenuates disease severity in models of proteotoxic Charcot-Marie-Tooth type 1B [Preprint] February 2, 2024
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