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.

An Extraordinary Year and the Road Ahead

Jan 3, 2024 | Press Releases

As we begin 2024, I want to take a look back and celebrate the extraordinary year we had at the CMT Research Foundation. Since 2018, CMTRF has had an outsized impact on inspiring drug hunters to work on developing therapeutics for CMT and establishing itself as a key...

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...

Dr. Grace Pavlath Joins CMT Research Foundation’s Research Advisory Committee

Nov 14, 2023 | Press Releases

ATLANTA (November 14, 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 that Dr. Grace Pavlath has joined its newly formed Research Advisory Committee...

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...

Arthur Suckow, former Co-founder and Chief Executive Officer of DTx Pharma, Joins CMT Research Foundation Board of Directors

Oct 3, 2023 | Press Releases

ATLANTA (October 3, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease*, today announced that Arthur Suckow, Ph.D. former Co-founder and Chief Executive Officer of DTx...

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...

The CMT Research Foundation Launches Annual CMT Action Month with Special September of Events

Sep 1, 2023 | Press Releases

To mark CMT Action Month this September, the CMT Research Foundation (CMTRF) today launched a month-long lineup of events, bringing together artists, advocates, and the CMT community from across the U.S. The series of events aim to raise awareness and critical funds...