Project Testing PIKfyve Inhibitor in CMT4B1 Clears First Hurdles
By: Keith Fargo, PhD, Chief Scientific Officer We are 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...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...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...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...
Important Research News Announcements
- The phenotypic spectrum of pathogenic ATP1A1 variants expands: the novel p.P600R substitution causes demyelinating Charcot-Marie-Tooth disease February 4, 2023
- Diverse CMT2 neuropathies are linked to aberrant G3BP interactions in stress granules February 3, 2023
- Retraction: Charcot-Marie-Tooth Disease as a Risk Factor for Periprosthetic Fractures in Tibiotalocalcaneal Fusion With Intramedullary Nailing January 20, 2023
- INF2 mutations in patients with a broad phenotypic spectrum of Charcot-Marie-Tooth disease and focal segmental glomerulosclerosis January 13, 2023
- Exercise induced myelin protein zero improvement in neuropathic pain rats January 11, 2023
- A meta-analysis on the prevalence of Charcot-Marie-Tooth disease and related inherited peripheral neuropathies January 11, 2023
- X-linked Charcot-Marie-Tooth disease after SARS-CoV-2 vaccination mimicked stroke-like episodes: A case report January 11, 2023
- Targeting the PMP22 Protein to Develop Leads Against Charcot-Marie-Tooth Disease December 31, 2022
- Actin-mediated regulation of organelle dynamics in Charcot-Marie-Tooth disease December 31, 2022
- Electroceutical approach ameliorates intracellular PMP22 aggregation and promotes pro-myelinating pathways in a CMT1A in vitro model December 30, 2022