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 to Benefit from a Portion of Ticket Sales from Alan Jackson’s Last Call: One More for the Road – The “Finale”
Alan Jackson is ready to serve up one more round before he retires from touring—and he’s inviting a few friends to join him! Last Call: One More for the Road - The Finale – the last full-length concert of Jackson’s touring career – will take place Saturday, June 27,...
Dr. Meghan Drummond Joins the CMT Research Foundation as Vice President of Research and Drug Development
Dr. Meghan Drummond, PhD, has joined the CMT Research Foundation as vice president of research and drug development. She will lead the Foundation’s research and drug development strategy, overseeing the advancement of promising therapeutic approaches for...
Applied Therapeutics to Meet With FDA on New Drug Application for the Treatment of CMT-SORD
Applied Therapeutics, who will be speaking at the 2025 Global CMT Research Convention, recently announced that they will be meeting with the U.S. Food and Drug Administration, during the third quarter of 2025, to discuss a potential New Drug Application for...
Dr. Yulia Grishchuk Joins CMTRF’s Scientific Advisory Board
CMT Research Foundation is pleased to announce that Dr. Yulia Grishchuk, Ph.D., 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...
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...
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...
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...