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.
CMT Research Foundation Invests in XtRNA Bio to Develop Gene Therapy for CMT2D
CMT Research Foundation has invested in a research project with XtRNA Bio aimed to develop a new viral gene therapy for Charcot-Marie-Tooth disease type 2D. How GARS1 Mutations Drive CMT2D CMT2D is caused by mutations in the GARS1 gene, which encodes glycyl-tRNA...
A Father Who Defies ‘Impossible’: Joao Cardoso’s Journey with CMT
Joao Cardoso, Brittany, France, Living with CMT Joao Cardoso never imagined that he would be riding through the town of Brittany, France, in a wheelchair, while his eight-year-old son, Edgar, pedals his bike alongside him. Because of Joao’s Charcot-Marie-Tooth...
CMT Research Foundation Invests in Asha Therapeutics to Test Novel Drug for CMT2A
CMT Research Foundation has invested in a research project with Asha Therapeutics to investigate the therapeutic potential of inhibiting SARM1 (Sterile alpha and TIR motif-containing protein 1) using their novel drug, ASHA-624, for the treatment of Charcot-Marie-Tooth...
Vanderbilt University Researchers Identify Promising Compounds Targeting PMP22 Protein Expression, Paving the Way for New Treatments for CMT1A, CMT1E and HNPP
Researchers at Vanderbilt University have made significant progress in the development of small molecules aimed at treating Charcot-Marie-Tooth disease types caused by mutations in the PMP22 gene, which include CMT1A, CMT1E and hereditary neuropathy with liability to...
Because of Luca: A Family’s Fight Against CMT
Tara, Luca, Liam and Lane, Louisiana, Living With CMT When Luca Haik was just four years old, his mother, Tara, sensed something wasn’t right. “Luca came home from school every day crying in pain, rushing to take off his socks and shoes saying that his feet ‘felt...
82VS Progressing Ahead of Schedule in CMTRF-Funded Project to Improve Oligonucleotide Drug Delivery for CMT
Since launching their collaboration with CMT Research Foundation in November 2024, researchers at 82VS, the venture studio of Alloy Therapeutics, have made rapid progress and are ahead of schedule testing their novel technology focused on improving RNA therapeutic...
Initial CMTRF Funding Leads Augustine Therapeutics to Raise $85 Million for CMT Clinical Trials
CMT Research Foundation funded partner Augustine Therapeutics recently announced that they completed their Series A financing round and raised $85 million, which will be used to advance their Charcot-Marie-Tooth disease drug candidate, AGT-100216, through a Phase I/II...
More Than Just a Rare Disease: Jodi Balog’s Journey With CMT
Jodi Balog, Pennsylvania, Living With CMT Jodi Balog has lived with Charcot-Marie-Tooth disease for more than 30 years. Diagnosed at the age of 13, CMT has become all she’s ever known. While CMT is classified as a rare disease, for Jodi — and millions of others — it’s...
The Greatest Gift: Jillian Cabernel’s Story of Strength, Magic and the Hope for a Cure
The Greatest Gift: Jillian Cabernel, Vancouver, BC, Living With CMT December 2023 was a Christmas to remember for Jillian Cabernel. For the first time in her life, she truly felt the magic of the holidays. Jillian has spent her entire life living with...
CMT Research Foundation Funds Project with Alloy Therapeutics’ 82VS to Test Novel RNA Therapeutics for Charcot-Marie-Tooth Disease
The CMT Research Foundation has invested over $500,000 in a program led by 82VS, the venture studio of Alloy Therapeutics, Inc. to discover and develop novel Antibody Oligonucleotide Conjugate (AOC) therapeutics for CMT. Oligonucleotide therapeutics offer promise for...