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

NCT07226297 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

ENROLLING_BY_INVITATION

Status

1

Enrollment

OTHER

Sponsor class

Conditions

Charcot-Marie-Tooth Disease Type 2D

Interventions

DRUG: nL-GARS1-001

Sponsor

n-Lorem Foundation

Collaborators

[object Object]