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.

CMT Research Foundation and Nanite Inc. Announce Partnership to Enhance Therapeutic Efficacy of Antisense Oligonucleotides in CMT1A

Feb 22, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

ATLANTA, GA (February 22, 2023) The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced a partnership with Nanite, Inc., a Boston-based biotechnology company developing...

Continuation of Inflammation Research for CMT1B

Feb 14, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

In 2020, the CMT Research Foundation had funded renowned nerve disease expert, Dr. Rudolf Martini of University Hospital Würzburg (Germany), to investigate whether inhibiting inflammation in the peripheral nerves could reduce symptoms and improve outcomes for patients...

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...

2022 at CMTRF

Jan 9, 2023 | CMT Research Updates, CMTRF Funded Research, Press Releases

Dear Friends of CMTRF, In preparing for another busy year at CMTRF, I took some time recently to look back at the accomplishments of 2022. I am immensely proud of what our team has achieved (summarized below) and I believe it underscores how CMTRF adds significant...

Dr. Arthur Suckow, DTx Pharma Inc. – FALCON

Nov 28, 2022 | CMTRF Funded Research, Drug Development

One of the first research teams the CMT Research Foundation (CMTRF) funded was DTx Pharma, Inc., and the project focused on delivery of genetic medicine to the myelinating Schwann cells of the peripheral nerves - a major scientific challenge to effectively treating...

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...

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...