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.
Dr. Afrooz Rashnonejad Completes Initial Studies Demonstrating the Effectiveness of a Novel Gene Therapy for CMT1B
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
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
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
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
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
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
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
Novartis acquires DTx’s FALCON technology to advance treatment development for CMT1A
Joao Cardoso, Paralympic Table Tennis Player with CMT2A
Meet Joao Cardoso, a paralympic table tennis player from Portugal, living in France, and competitor in this year’s International Para Championship. In his 5 years of competing, Joao has earned many awards and titles, including the Medal of Sporting Merit and this...
Dr. William W. Motley to Join CMT Research Foundation Board of Directors
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...