Active, not recruitingPHASE1, PHASE2NCT07197268

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation

Studying Bainbridge-Ropers syndrome

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Key facts

Sponsor
n-Lorem Foundation
Intervention
nL-ASXL3-001(drug)
Enrollment
1 enrolled
Eligibility
4-4 years · MALE
Timeline
20252027

Study locations (1)

Collaborators

University of North Carolina, Chapel Hill

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 NCT07197268 on ClinicalTrials.gov

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