Research News and Updates
Read the latest from the CMT Research Foundation on drug development and clinical advances.
CMT Research Foundation partners with Samsara Therapeutics to develop a novel class of drugs in CMT1A
By: Keith Fargo, PhD, Chief Science Officer The CMT Research Foundation is pleased to announce a new project with Samsara Therapeutics, a US/UK-based biotech company that is developing a novel class of drugs for CMT1A. CMT1A is caused by duplication of a stretch of...
CMT Research Foundation and Samsara Therapeutics Partner on a Novel Therapeutic Approach for CMT1A with the Potential to Reach Clinical Trials
ATLANTA (October 13, 2022) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease*, has invested in Samsara Therapeutics Inc., a biotech company focusing on discovering and developing...
CMT Research Foundation Awards $152,524 to ORYZON to test novel HDAC6 inhibitors in CMT1A
By: Keith Fargo, PhD, Chief Science Officer New project will examine impact of two selective and potent drug candidates recently discovered by ORYZON The CMT Research Foundation is delighted to announce a new project with ORYZON, a clinical stage pharmaceutical...
ORYZON collaborates with the CMT Research Foundation in the US
CMTRF to provide funding for in vivo efficacy tests of Oryzon’s HDAC6 inhibitors in Charcot-Marie-Tooth preclinical model MADRID, SPAIN and Atlanta, July 26, 2022 Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company...
The CMT Research Foundation Invests Over $500,000 to Find a Single Gene Therapy for Possibly Every Form of CMT1B
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
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
The CMT Research Foundation meets with more than 30 companies at BIO and is recognized as a leader in patient driven research.
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...
Important Research News Announcements
- Charcot-Marie-Tooth neuropathies: current gene therapy advances and the route towards translation March 25, 2023
- MFN2-dependent recruitment of ATAT1 coordinates mitochondria motility with alpha-tubulin acetylation and is disrupted in CMT2A March 17, 2023
- Boosting peripheral BDNF rescues impaired in vivo axonal transport in CMT2D mice March 16, 2023
- Restoring Neurofilaments to Axons in a Mouse Model of CMT2E March 1, 2023
- Full-length isoform sequencing for resolving the molecular basis of Charcot-Marie-Tooth 2A February 8, 2023
- Torin1 restores proliferation rate in Charcot-Marie-Tooth disease type 2A cells harbouring MFN2 (mitofusin 2) mutation February 3, 2023
- Novel Variants in MPV17, PRX, GJB1 , and SACS Cause Charcot-Marie-Tooth and Spastic Ataxia of Charlevoix-Saguenay Type Diseases January 30, 2023
- Pharmacologic Targeting of the C-Terminus of Heat Shock Protein 90 Improves Neuromuscular Function in Animal Models of Charcot Marie Tooth X1 Disease January 20, 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
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