Small Molecule Therapies for CMT

Small molecule therapies typically are developed from simple building blocks or are derived from natural sources. With their small size, they are easily able to pass through the cell membrane and access targets inside the cell.

Small molecule drugs are designed to modify a disease process via regulation of a biological target such as an enzyme, channel or receptor. They make up the majority of today’s drug treatments and may be used alone or as individual elements in combination therapies.

CMT Research Foundation is leading the charge to ensure safe and effective therapies for all forms of CMT make it to the market to help those living with CMT today. We are working every day to support the families, the foundations, the academics, the scientists, the biotechs and the pharma companies who share our goal and who are working to make it happen.

CMT Research Foundation Launches Research Advisory Committee of Renowned Scientific Experts

by George Simpson | Oct 18, 2023 | Foundation News, 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

by Chelsea Layton | Oct 3, 2023 | Foundation News

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...

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

by Kayleena Speakman | 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

by George Simpson | 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

by Chelsea Layton | 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

by George Simpson | Sep 1, 2023 | Foundation News

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

by George Simpson | Aug 14, 2023 | Foundation News

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

by Chelsea Layton | Jul 19, 2023 | Foundation News

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

by Chelsea Layton | 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...