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.
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...
Siemens’ Ann Fairchild Joins CMT Research Foundation Board of Directors
CMT Research Foundation is pleased to announce that Ann Fairchild, executive vice president and general counsel of Siemens USA, has joined the Foundation’s Board of Directors. Fairchild is a seasoned legal executive with more than 25 years of experience at Siemens,...
Applied Therapeutics Presents Findings From Phase 2/3 Clinical Trial of Govorestat in CMT-SORD
Applied Therapeutics recently presented findings from their INSPIRE Phase 2/3 clinical trial of govorestat (AT-007) for the treatment of Sorbitol Dehydrogenase Deficiency, a subtype of Charcot-Marie-Tooth disease, at the Peripheral Nerve Society’s 2025 Annual Meeting...
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...
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...
OHio, What a Night: Lighting a Path to a Cure for Charcot-Marie-Tooth Disease
On Thursday, April 10, more than 120 patients, scientists, clinicians and pharma companies gathered in Columbus, Ohio for OHio, What a Night — an unforgettable evening dedicated to accelerating research and developing treatments and cures for Charcot-Marie-Tooth...
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...
Dr. Afrooz Rashnonejad Joins CMTRF’s Scientific Advisory Board
CMT Research Foundation is pleased to announce that Dr. Afrooz Rashnonejad has joined the Foundation’s Scientific Advisory Board. The SAB is composed of distinguished scientific and clinical experts specializing in Charcot-Marie-Tooth disease and drug development. The...
NMD Pharma Announces FDA Orphan Drug Designation for NMD670
NMD Pharma has announced that they have been granted orphan drug designation by the Food and Drug Administration for NMD670, their novel, oral, small molecule inhibitor of the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of...