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.
Applied Therapeutics Presents Findings From Phase 2/3 Clinical Trial of Govorestat in CMT-SORD
Applied Therapeutics recently presented findings from their INSPIRE Phase 2/3 clinical trial of govorestat (AT-007) for the treatment of Sorbitol Dehydrogenase Deficiency, a subtype of Charcot-Marie-Tooth disease, at the Peripheral Nerve Society’s 2025 Annual Meeting...
CMT Research Foundation Invests in Asha Therapeutics to Test Novel Drug for CMT2A
CMT Research Foundation has invested in a research project with Asha Therapeutics to investigate the therapeutic potential of inhibiting SARM1 (Sterile alpha and TIR motif-containing protein 1) using their novel drug, ASHA-624, for the treatment of Charcot-Marie-Tooth...
Initial CMTRF Funding Leads Augustine Therapeutics to Raise $85 Million for CMT Clinical Trials
CMT Research Foundation funded partner Augustine Therapeutics recently announced that they completed their Series A financing round and raised $85 million, which will be used to advance their Charcot-Marie-Tooth disease drug candidate, AGT-100216, through a Phase I/II...
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...
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...
Actio Biosciences Receives Both Orphan Drug and Rare Pediatric Disease Designations From the FDA For Treatment of CMT2C
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...
NMD Pharma Receives FDA Clearance to Initiate Clinical Trial of NMD670 for CMT
NMD Pharma recently announced that it has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 2 clinical trial of NMD670 in patients living with CMT types 1 and 2. NMD670 is a...
CMT Research Foundation Invests in Project to Test if a Commercially Available Drug is Effective Treatment for X-linked Charcot-Marie-Tooth Disease
The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT), has invested in a project led by Dr. Charles Abrams, a Professor in the Department of Neurology and Rehabilitation at the...
CMT Research Update/NMD Pharma
NMD Pharma recently published data demonstrating that their drug candidate, NMD670, improves muscle function and neuromuscular transmission deficits in both animal models and patients. Dr. William David Arnold, a member of the CMTRF Scientific Advisory Board, who was...
Updates on CMT Research
There have been a number of interesting developments in CMT research in the past few weeks. Here are three: Update 1: Pharnext recently announced topline results of its pivotal phase 3 (PREMIER trial) of PXT3003, its drug candidate for Charcot-Marie-Tooth 1A (CMT1A)...