Research News and Updates
Read the latest from the CMT Research Foundation on drug development and clinical advances.
2022 Global CMT Research Convention—Introducing the Scientific Program Committee
By Keith Fargo, Chief Scientific Officer 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...
Shift Pharmaceuticals Completes CMT Research Foundation-Funded Project, Will Continue Development of Gene Therapy for CMT1A
By Keith Fargo, Chief Scientific Officer 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...
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
By Keith Fargo, Chief Scientific Officer 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...
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. By: Susan Ruediger, Co Founder and Chief Mission Officer With the recent opening of their new NextGen Precision Health Institute, University of Missouri...
AcuraStem Completes CMT Research Foundation-Funded Project, Finds Eleven Potential Therapeutics for CMT2A
By Keith Fargo, Chief Scientific Officer 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....
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. By Keith Fargo, Ph.D., Chief Scientific Officer, CMT Research FoundationThe CMT Research Foundation is pleased to announce that Dr. Kleopas Kleopa, a renowned neuromuscular...
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...
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...
Important Research News Announcements
- 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
- The Regentime stem cell procedure, successful treatment for a Charcot-Marie-Tooth disease case December 28, 2023
- Preclinical Efficacy of Peripheral Nerve Regeneration by Schwann Cell-like Cells Differentiated from Human Tonsil-Derived Mesenchymal Stem Cells in C22 Mice December 18, 2023
- AAV-mediated editing of PMP22 rescues Charcot-Marie-Tooth disease type 1A features in patient-derived iPS Schwann cells November 28, 2023
- Combined RNA interference and gene replacement therapy targeting MFN2 as proof of principle for the treatment of Charcot-Marie-Tooth type 2A November 25, 2023
- Mouse models of human CNTNAP1-associated congenital hypomyelinating neuropathy and genetic restoration of murine neurological deficits October 19, 2023
- Intravenous Administration of an AAV9 Vector Ubiquitously Expressing C1orf194 Gene Improved CMT-Like Neuropathy in C1orf194-/- Mice October 16, 2023
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