News & Stories

See the latest news about CMT drug development and read stories from the CMT community that highlight why we must deliver treatments and cures during our lifetime.

Optimal Dosing Identified in CMT1X Research Update

Jan 31, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

A CMT Research Foundation-funded project at the University of Illinois Chicago has identified optimal dosing for a potential therapeutic for CMT1X. Like other forms of CMT, cell-to-cell communication within the peripheral nervous system malfunctions in people with...

CMT2E Gene Therapy Project Successfully Completes Milestone 2

Jan 17, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

A collaboration between Dr. Chris Lorson and Dr. Michael Garcia of the University of Missouri and funded by the CMT Research Foundation is producing promising results in an innovative new approach to treating CMT2E. CMT2E presents a special challenge in that the...

Italian Research Group Completes CMT Research Foundation-Funded Project, Results Encourage Further Work in New Therapy for Adult and Elderly Patients with CMT1A/CMT1B

Jan 10, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

The CMT Research Foundation is pleased to announce that Dr. Maurizio D'Antonio's research team at San Raffaele Scientific Institute in Milan, Italy has completed their CMT Research Foundation-funded project investigating the role of a protein called Gadd34 in a mouse...

CMT Research Foundation partners with Samsara Therapeutics to develop a novel class of drugs in CMT1A

Oct 13, 2022 | About CMT, CMT Research Updates, CMTRF Funded Research, Research News

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 DNA that includes the PMP22 gene, and people...

CMT Research Foundation and Samsara Therapeutics Partner on a Novel Therapeutic Approach for CMT1A with the Potential to Reach Clinical Trials

Oct 12, 2022 | CMTRF Funded Research, Press Releases, Research News

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

Jul 26, 2022 | CMTRF Funded Research, Research News

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 company based in Spain. Histone deacetylase (HDAC)...

ORYZON collaborates with the CMT Research Foundation in the US

Jul 26, 2022 | CMTRF Funded Research, Press Releases, Research News

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

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

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 of the nerves that facilitate rapid...

Project Testing PIKfyve Inhibitor in CMT4B1 Clears First Hurdles

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

CMTRF is 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 preclinical trial in CMT4B1 model mice. Before the...

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

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 molecules designed to reduce the expression of...