U.S., Oct. 30 -- ClinicalTrials.gov registry received information related to the study (NCT07222371) titled 'An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy' on Oct. 27.
Brief Summary: This current study is aimed for the treatment of an individual participant with a form of TUBB4A-related leukodystrophy with hypomyelination.
Study Start Date: Sept. 19
Study Type: INTERVENTIONAL
Condition:
Genetic Disease
TUBB4A-Related Leukodystrophy
Intervention:
DRUG: nL-TUBB4-001
Personalized antisense oligonucleotide
Recruitment Status: ACTIVE_NOT_RECRUITING
Sponsor: University of California, San Diego
Information provided by (Responsible Party): O...