Charcot-marie-tooth Disease Type 2d

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CLINICAL_TRIALS research · 2025-11-10
Clinical Trial Update: Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D) [Phase 1, Phase 2]
Ongoing Phase 1/2 clinical trial of a personalized antisense oligonucleotide for Charcot-Marie-Tooth disease Type 2D caused by GARS1 mutatio…