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.

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...

NMD Pharma Receives FDA Clearance to Initiate Clinical Trial of NMD670 for CMT

Jun 18, 2024 | CMT Research Updates, Drug Development

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

Apr 23, 2024 | CMT Research Updates, CMTRF Funded Research, Press Releases, Research News

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

Apr 1, 2024 | CMT Research Updates, Drug Development, Research News

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

Dec 21, 2023 | CMT Research Updates, Research News

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)...

Update from the Novartis CMT1A Development Team

Oct 29, 2023 | About CMT, Drug Development, News

As you know, the CMT Research Foundation is proud to have introduced DTx leadership to Charcot-Marie-Tooth disease in 2019. We funded their first work in CMT to demonstrate the FALCON platform's potential to deliver a drug to the Schwann cells. And from there, the...

ARM-101: A Potential “Precision Drug Therapy” For CMT1A

Oct 24, 2023 | CMTRF Funded Research, Research News

The CMT Research Foundation has invested in a project at Armatus Bio, Inc. that will advance a novel miRNA gene therapy candidate for CMT type 1A. Principal Investigator Dr. Brian Price and his colleagues aim to harness the potential of microRNAs (miRNAs), snippets of...

CMT Research Foundation Launches Research Advisory Committee of Renowned Scientific Experts

Oct 18, 2023 | Press Releases, Research News

ATLANTA (October 18, 2023) The CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced the formation of a Research Advisory Committee (RAC) consisting of: Dr. William...

Oryzon Develops Novel HDAC6 Inhibitor ORY-4001 Showing Therapeutic Potential for CMT in Mouse Model

Jul 18, 2023 | CMT Research Updates, CMTRF Funded Research, Research News

Scientists have been studying a potential treatment option for CMT by inhibiting (blocking) a protein called histone deacetylase 6 (HDAC6). They have found that HDAC6 plays an important role in regulating the pathways involved in breaking down proteins and...