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Clinical trial

An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Charcot-Marie-Tooth Type 2D Due to GARS1 Genetic Mutation

NCT07226297 is registered in ClinicalTrials.gov with n-Lorem Foundation as lead sponsor.

NCT NCT07226297
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Charcot-Marie-Tooth Type 2D Due to GARS1 Genetic Mutation
Lead sponsor
n-Lorem Foundation
Phase
Phase 1/2
Overall status
Enrolling by invitation
Start date
October 27, 2025
Completion date
October 31, 2027
Enrollment
1 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

charcot-marie-tooth disease type 2d