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.

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)...

The Importance of Fundraising

Nov 14, 2023 | About CMT, Life with CMT

By: Miron Hall Because CMT is a rare disease, it often receives less attention and funding than more common medical conditions. Without adequate resources, researchers struggle to develop effective treatments and ultimately find a cure. This is where fundraising plays...

Update from the Novartis CMT1A Development Team

Oct 29, 2023 | About CMT, Drug Development

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 Invests in Armatus Bio, Inc. to Advance Potential Therapeutic for CMT1A

Oct 23, 2023 | CMT Research Updates, CMTRF Funded Research

ATLANTA (October 24, 2023) The CMT Research Foundation, a non-profit focused solely on delivering treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has invested in a project at Armatus Bio that will advance a novel miRNA gene therapy candidate for CMT type...

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...

Dr. Afrooz Rashnonejad Completes Initial Studies Demonstrating the Effectiveness of a Novel Gene Therapy for CMT1B

Sep 30, 2023 | CMTRF Funded Research

Charcot-Marie-Tooth type 1B is caused by mutations myelin protein zero, which is an essential component making myelin, the protective layer or sheath around nerves. Defective myelin protein zero results in damaged or impaired myelin leading to progressive nerve damage...

Another Asset with Potential to Treat Charcot-Marie-Tooth has been Acquired by Pharma

Sep 26, 2023 | CMT Research Updates, CMTRF Funded Research, Drug Development, Research News

An asset that is being tested in CMT4B1 underwritten by the CMT Research Foundation has been licensed by AcuraStem to Takeda for approximately $580 million. The exclusive, worldwide license will enable Takeda to develop and commercialize AcuraStem's PIKFYVE targeted...

Endgame Campaign for CMT1A Tops $6.6 Million

Sep 20, 2023 | CMT Research Updates, CMTRF Funded Research

Recently, Peter J. deSilva, CMTRF Board member and Chair of the ENDGAME Capital Campaign to end CMT1A and Susan Ruediger, CMT patient, co-founder, and Chief Mission Officer of the CMTRF, hosted a private call with donors to update them on the progress being made in...

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...