


Dr. Arthur Suckow, DTx Pharma Inc. – FALCON
One of the first research teams the CMT Research Foundation (CMTRF) funded was DTx Pharma, Inc., and the project focused on delivery of genetic medicine to the myelinating Schwann cells of the peripheral nerves - a major scientific challenge to effectively treating...

A New Trial for CMT – When Science Moves Quickly
By Keith Fargo, Ph.D., Chief Scientific Officer, CMT Research Foundation The pace of science can seem excruciatingly slow. It can take many years, or even decades, for new ideas to result in clinical trials. Every so often, though, the stars align and a new...

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
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
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...

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

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