Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
University of California, San Diego
The study is an investigational interventional drug created for the study subject's specific pathogenic genetic mutation.
Age
Any age
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
Participant has any known contraindication to or unwillingness to undergo lumbar puncture
Use of investigational medication within 5 half-lives of the drug at enrollment
Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.
Clareo Health | An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy
