Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

NCT06369974 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

ENROLLING_BY_INVITATION

Status

1

Enrollment

OTHER

Sponsor class

Conditions

Genetic Disease

Interventions

DRUG: Antisense oligonucleotide treatment (ASO)

Sponsor

Massachusetts General Hospital

Collaborators

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