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.

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

Sep 26, 2023 | News, Press Releases

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...

W. David Arnold, MD Joins CMT Research Foundation Scientific Advisory Board

Aug 14, 2023 | News, Press Releases

ATLANTA (August 14, 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 Dr. W. David (Dave) Arnold has joined the Foundation’s Scientific Advisory...

Bipasha Mukherjee-Clavin, MD, PhD and Corey Hopkins, PhD Join CMT Research Foundation Scientific Advisory Board

Jul 19, 2023 | Press Releases

ATLANTA (July 17, 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 two world-class researchers have joined its Scientific Advisory Board (SAB). Dr....

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...

DTx Pharma Acquired by Novartis for CMT1A Therapy

Jul 17, 2023 | CMTRF Funded Research, Drug Development, Research News

Novartis acquires DTx’s FALCON technology to advance treatment development for CMT1A

Dr. William W. Motley to Join CMT Research Foundation Board of Directors

Jun 21, 2023 | Press Releases

ATLANTA (June 21, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth disease*, today announced that Dr. William W. Motley, a neurologist with a doctorate in neurogenetics with fifteen...

DTx Pharma

May 8, 2023 | About CMT, News

We are pleased share this story that published on May 3: https://patientworthy.com/2023/05/03/dtx-pharma-developing-cmt1a-rna-therapeutics/ One of the early programs CMTRF funded was DTx, a young company that had developed a new platform for RNA therapeutics. DTx was...