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.

The Greatest Gift: Jillian Cabernel’s Story of Strength, Magic and the Hope for a Cure

Nov 18, 2024 | Life with CMT

The Greatest Gift: Jillian Cabernel, Vancouver, BC, Living With CMT December 2023 was a Christmas to remember for Jillian Cabernel. For the first time in her life, she truly felt the magic of the holidays. Jillian has spent her entire life living with...

Update on NMD Pharma Clinical Trial

Nov 18, 2024 | CMT Research Updates

NMD Pharma, a clinical-stage biotech company dedicated to developing novel and improved treatments for patients living with neuromuscular diseases, has announced that it has dosed the first Charcot-Marie-Tooth disease patient in its Phase 2a clinical trial of NMD670...

CMT Research Foundation Funds Project with Alloy Therapeutics’ 82VS to Test Novel RNA Therapeutics for Charcot-Marie-Tooth Disease

Nov 13, 2024 | CMT Research Updates, CMTRF Funded Research

The CMT Research Foundation has invested over $500,000 in a program led by 82VS, the venture studio of Alloy Therapeutics, Inc. to discover and develop novel Antibody Oligonucleotide Conjugate (AOC) therapeutics for CMT. Oligonucleotide therapeutics offer promise for...

Dr. Alessandra Bolino Completes Project Testing PIKfyve Inhibitor Drug from AcuraStem in CMT4B1 models

Nov 1, 2024 | CMT Research Updates, CMTRF Funded Research

Previous research from Dr. Alessandra Bolino demonstrated that inhibiting a protein called PIKfyve kinase could ameliorate some disease characteristics in CMT4B1 cells. In this project, Dr. Bolino partnered with AcuraStem to test one of their PIKfyve inhibitor drug...

Actio Biosciences Receives Both Orphan Drug and Rare Pediatric Disease Designations From the FDA For Treatment of CMT2C

Aug 9, 2024 | CMT Research Updates, Drug Development

Actio Biosciences recently announced that they have received both orphan drug designation and rare pediatric disease designation for ABS-0871, a TRPV4 inhibitor, for the treatment of TRPV4+ Charcot-Marie-Tooth disease subtype 2C (CMT2C) from the U.S. Food and Drug...

CMT Research Foundation Invests in Project at Nationwide Children’s Hospital to Develop Improved Gene Therapy Delivery Systems for Several Types of CMT

Aug 1, 2024 | CMTRF Funded Research

The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has invested in a research project to develop new vehicles for delivering gene therapies more efficiently into peripheral nerves. This...

Armatus Bio Completes Pivotal Animal Study Demonstrating Delivery, Safety, and Activity of Their CMT1A Gene Therapy and is Now Preparing to Advance Project to Clinical Trials

Jul 30, 2024 | CMT Research Updates, CMTRF Funded Research

Armatus Bio acquired the CMT1A gene therapy that was developed by Dr. Kleopas Kleopa and Dr. Scott Harper in a previous CMTRF-funded project to advance it further as a therapeutic. To demonstrate this therapy could be a safe and effective treatment for CMT1A, Armatus...

Inaugural Zero-K Run Raises Awareness for CMT and CMTRF

Jul 19, 2024 | Life with CMT

When many people think of the word “race” they imagine a 5k, 10k and even a half-marathon. But, what about a new type of run that directly benefits patients with Charcot-Marie-Tooth disease and covers no distance at all—A Zero-K Run. A Zero-K Run is exactly what it...

Augustine Therapeutics Raises $18.5 Million in a Series A First Closing

Jun 26, 2024 | CMT Research Updates, CMTRF Funded Research

The Charcot-Marie-Tooth Research Foundation participated in the $18.5 million Series A funding round recently announced by Augustine Therapeutics. The financing was led by Asabys Partners, with the participation of Eli Lilly & Company, CMTRF, and current investors...

CMT Research Foundation Invests in ReviR Therapeutics Research to Pioneer Small Molecule Therapeutics for CMT1A

Jun 19, 2024 | CMT Research Updates, CMTRF Funded Research

The CMT Research Foundation has formed a strategic partnership with ReviR Therapeutics to advance small molecule therapeutics that modulate the expression of the gene that causes CMT1A. ReviR Therapeutics is developing a class of drugs called small molecule splice...