An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

NCT07222371 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

ACTIVE_NOT_RECRUITING

Status

1

Enrollment

OTHER

Sponsor class

Conditions

Genetic Disease
TUBB4A-Related Leukodystrophy

Interventions

DRUG: nL-TUBB4-001

Sponsor

University of California, San Diego