Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Autosomal Dominant Leukodystrophy (ADLD) due to LMNB1 mutation

Inclusion Criteria: * Informed consent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s). * Autosomal dominant adult-onset leukodystrophy (ADLD) caused by an LMNB1 duplication mutation * 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. * Willingness to follow contraceptive guidance during the intervention period and for at least 40 weeks after the last dose of study intervention Exclusion Criteria: * Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures