In a recent announcement, Vanda Pharmaceuticals Inc. shared that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for its VCA-894A compound. This approval marks a significant step forward for community members who have ...
In a statement released on February 15, 2024, by Charcot-Marie-Tooth Association (CMTA) Alliance Partner, Applied Therapeutics, the company said that its INSPIRE Phase III trial for a certain type of Charcot-Marie-Tooth disease (CMT), called SORD-deficiency, showed positive interim 12-month results ...
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 ...