Overview

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

Status:
ACTIVE_NOT_RECRUITING

Trial end date:
2027-09-01

Target enrollment:

Participant gender:

Summary

This current study is aimed for the treatment of an individual participant with a form of TUBB4A-related leukodystrophy with hypomyelination.

Phase:

PHASE1

Details

Lead Sponsor:

University of California, San Diego