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

U.S., Nov. 11 -- ClinicalTrials.gov registry received information related to the study (NCT07226297) titled 'Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)' on Nov. 04.

Brief Summary: 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

Study Start Date: Oct. 27

Study Type: INTERVENTIONAL

Condition: Charcot-Marie-Tooth Disease Type 2D

Intervention: DRUG: nL-GARS1-001

Personalized antisense oligonucleotide

Recruitment Status: ENROLLING_BY_INVITATION

Spons...