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

NCT07197268 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

ACTIVE_NOT_RECRUITING

Status

1

Enrollment

OTHER

Sponsor class

Conditions

Bainbridge-Ropers Syndrome

Interventions

DRUG: nL-ASXL3-001

Sponsor

n-Lorem Foundation

Collaborators

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