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.

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 | CMTRF Funded Research, Drug Development

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...

DTx Pharma Video – Using siRNA to Treat Charcot-Marie-Tooth Disease Type 1A

May 7, 2023 | CMTRF Funded Research, Drug Development, Research News

Thomas F. Sander Joins CMT Research Foundation Board of Directors

Apr 27, 2023 | Press Releases

ATLANTA (April 26, 2023) CMT Research Foundation (CMTRF), a patient-led, non-profit focused on delivering treatments and cures for Charcot-Marie-Tooth disease, today announced that rare disease sales specialist, Thomas F. Sander, has joined its Board of Directors....

Edritz Javelosa to Join the CMT Research Foundation as Chief Scientific Officer

Apr 17, 2023 | Press Releases

ATLANTA (April 17, 2023) Edritz Javelosa (Hav-a-low-sa), PhD, who has been at the Muscular Dystrophy Association (MDA) since 2019 and Research Portfolio Director since 2021, has joined the CMT Research Foundation (CMTRF) as Chief Scientific Officer. Reporting to...

The CMT Research Foundation Appoints Anna Combes Vice President of Philanthropy

Apr 10, 2023 | Press Releases

ATLANTA (April 10, 2023) Anna Combes has joined the CMT Research Foundation in the newly-created position of Vice President of Philanthropy. Reporting to the Chief Operating Officer, Ms. Combes will be responsible for fundraising and development activities, including...

The CMT Research Foundation (CMTRF) Joins the European CMT Federation (ECMTF)

Mar 28, 2023 | Press Releases

The CMTRF is happy to announce that they are joining the ECMTF, a non-profit organization grouping European and worldwide national associations supporting people affected by the Charcot-Marie-Tooth (CMT) disease or neuromuscular disorders. One of the primary goals of...