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

Inclusion Criteria

• •Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s).
• •Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records.
• •Clinical phenotype and neuroimaging consistent with a diagnosis of TUBB4A-related leukodystrophy
• •Documented genetic mutation in TUBB4A