News

The Charcot-Marie-Tooth Association (CMTA) is pleased to share exciting news from our CMTA-STAR Alliance Partner, Actio Biosciences. The company has received both orphan drug designation (ODD) and rare pediatric disease designation (RPDD) designation from the FDA for ABS-0871, a TRPV4 ...

The Charcot-Marie-Tooth Association (CMTA) is pleased to share exciting news from our CMTA-STAR Alliance Partner, Applied Therapeutics. The company plans to submit a New Drug Application (NDA) to the FDA for govorestat to treat CMT-SORD (aka SORD Deficiency), a significant ...

The Charcot-Marie-Tooth Association (CMTA) has announced a $240K investment in a groundbreaking gene replacement therapy for CMT4A, with potential applications for other types of CMT. This critical project is led by Xin Chen, MD, PhD, and CMTA Scientific Advisory Board ...

In a groundbreaking three-year joint project between the Charcot-Marie-Tooth Association (CMTA) and the Muscular Dystrophy Association (MDA), researchers led by Alexia Kagiava, PhD, at the Cyprus Institute of Neurology and Genetics are pioneering the use of nanoparticles to develop gene ...

Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common form of CMT, caused by having an extra copy of the PMP22 gene. This results in PMP22 overexpression, leading to excessive PMP22 protein in Schwann cells, which produce the protective myelin ...