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The CMT Research Foundation Invests Over $500,000 to Find a Single Gene Therapy for Possibly Every Form of CMT1B

Jul 18, 2022 | CMT Research Updates, CMTRF Funded Research, Research news

By: Keith Fargo, PhD, Chief Science Officer CMT1B is one of the most common forms of CMT. In CMT1B, the part of the nervous system that is dysfunctional is the myelin sheath of the peripheral nerves. Created by cells called Schwann cells, myelin wraps around the parts...

Project Testing PIKfyve Inhibitor in CMT4B1 Clears First Hurdles

Jul 11, 2022 | About CMT, CMT Research Updates, CMTRF Funded Research, Research news

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...

CMT Research Foundation emerging as a leader at BIO International Convention

Jun 29, 2022 | CMT Research Updates, Stories

Recently Cleary Simpson (CEO), Keith Fargo (Chief Scientific Officer) and Susan Ruediger (Co-founder and Chief Mission Officer) traveled to San Diego for the annual BIO International Convention. BIO (Biotechnology Innovation Organization) brings together thousands of...

Shift Pharmaceuticals Completes CMT Research Foundation-Funded Project, Will Continue Development of Gene Therapy for CMT1A

May 4, 2022 | CMT Research Updates, CMTRF Funded Research, News, Press Releases, Research news

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

Apr 11, 2022 | About CMT, CMT Research Updates

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...

Gene therapy project for CMT2E has completed Milestone 1 successfully.

Feb 23, 2022 | CMT Research Updates, CMTRF Funded Research, Research news

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...
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RSS Important Research News Announcements

  • Histidine supplementation can escalate or rescue HARS deficiency in a Charcot Marie Tooth Disease model September 27, 2022
  • Anxiety and depression in Charcot-Marie-Tooth disease: data from the Italian CMT national registry September 16, 2022
  • A Charcot-Marie-Tooth-Causing Mutation in HSPB1 Decreases Cell Adaptation to Repeated Stress by Disrupting Autophagic Clearance of Misfolded Proteins September 15, 2022
  • Effectiveness of a specific physical therapy program for Charcot-Marie-Tooth on sleep quality, pain perception, and nocturnal cramps: a pilot study September 15, 2022
  • Heterozygous Seryl-tRNA synthetase 1 variants cause Charcot-Marie-Tooth disease September 11, 2022
  • Mitofusin 2 mutation drives cell proliferation in Charcot-Marie-Tooth 2A fibroblasts August 22, 2022
  • TFG mutation induces haploinsufficiency and drives axonal Charcot-Marie-Tooth disease by causing neurite degeneration August 20, 2022
  • Implantable Electroceutical Approach Improves Myelination by Restoring Membrane Integrity in a Mouse Model of Peripheral Demyelinating Neuropathy August 17, 2022
  • Charcot-Marie-Tooth Disease as a Risk Factor for Periprosthetic Fractures in Tibiotalocalcaneal Fusion With Intramedullary Nailing August 15, 2022
  • CMT2A-linked mitochondrial hyperfusion-driving mutant MFN2 perturbs ER-mitochondrial associations and Ca2+ homeostasis August 4, 2022

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