News

The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth disease (CMT) research that’s aimed at bringing treatments and a cure to patients of this rare disease, is pleased to announce the appointment of Dr. Charlotte Sumner to its Strategy ...

Multisite and multiyear collaborative project led by a team of international researchers Advancing CMT Treatment with CRISPR-Cas9 and small molecule drug development GLENOLDEN, PA, February 7, 2024 – The Charcot-Marie-Tooth Association (CMTA) announced today a groundbreaking international research initiative investing over ...

Significant Research Investment Made in 2023: We are excited to share that in 2023, the CMTA has ongoing and committed investments of $5,000,00 in research dedicated to finding effective treatments and ultimately a cure for Charcot-Marie-Tooth disease. Support for Natural History Studies Drives ...

CMTA Alliance Partner ToolGen announced December 18, 2023 that it received Orphan Drug Designation (ODD) for its rare disease gene correction treatment, TGT-001, from the U.S. Food and Drug Administration (FDA). TGT-001 targets Charcot-Marie-Tooth (CMT), a rare genetic disease related ...

Published: December 11, 2023 French biopharmaceutical company Pharnext SA announced Dec. 10 that the initial results from its Phase III clinical trial (PREMIER trial) for CMT1A did not meet expectations. The company said in a news release that analyzing the results was ...