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.
More Than Just a Rare Disease: Jodi Balog’s Journey With CMT
Jodi Balog, Pennsylvania, Living With CMT Jodi Balog has lived with Charcot-Marie-Tooth disease for more than 30 years. Diagnosed at the age of 13, CMT has become all she’s ever known. While CMT is classified as a rare disease, for Jodi — and millions of others — it’s...
Dr. Afrooz Rashnonejad Joins CMTRF’s Scientific Advisory Board
CMT Research Foundation is pleased to announce that Dr. Afrooz Rashnonejad has joined the Foundation’s Scientific Advisory Board. The SAB is composed of distinguished scientific and clinical experts specializing in Charcot-Marie-Tooth disease and drug development. The...
NMD Pharma Announces FDA Orphan Drug Designation for NMD670
NMD Pharma has announced that they have been granted orphan drug designation by the Food and Drug Administration for NMD670, their novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of...
Maintaining Momentum: CMTRF’s Vision for 2025 and Beyond
Greetings and Happy New Year to you all. When I began my tenure as CEO of the CMT Research Foundation in October 2024, I was immediately inspired and impressed by the work of the Foundation. I was equally moved by the dedication of the greater CMT community,...
NMD Pharma Publishes Findings Supporting the Role of ClC-1 Inhibition in Charcot-Marie-Tooth Disease
NMD Pharma announced that they have published new clinical and preclinical data that provides evidence that targeting neuromuscular junction deficits could potentially improve muscle function in CMT patients. The peer-reviewed paper titled “Neuromuscular Transmission...
CMTRF Earns Top Rating From Charity Navigator
The CMT Research Foundation has been evaluated for the first time by Charity Navigator, the nation’s largest charity evaluator, and earned a Four-Star Rating, the highest possible rating. Charity Navigator’s impartial, third-party evaluation validates CMTRF’s...
The Greatest Gift: Jillian Cabernel’s Story of Strength, Magic and the Hope for a Cure
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
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
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
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...
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