Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

Authors
Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2025
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Charcot-Marie-Tooth disease type 2D (CMT2D) due to a pathogenic, de novo deletion mutation in GARS1
Epistemonikos ID: 6bcc7a1f5a85e5dcd83a83b08027fb03e0d3a135
First added on: Nov 11, 2025