Enrolling by invitationPHASE1, PHASE2NCT07226297

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

Studying Autosomal dominant Charcot-Marie-Tooth disease type 2D

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Key facts

Sponsor
n-Lorem Foundation
Intervention
nL-GARS1-001(drug)
Enrollment
1 enrolled
Eligibility
13 years · FEMALE
Timeline
20252027

Study locations (1)

Collaborators

The University of Texas Health Science Center, Houston

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Open NCT07226297 on ClinicalTrials.gov
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