U.S., May 16 -- ClinicalTrials.gov registry received information related to the study (NCT07588581) titled 'Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation' on May 06.
Brief Summary: This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Childhood-Onset Neurodegeneration with Brain Atrophy (CONDBA) due to a heterozygous missense gain-of-function mutation in UBTF
Study Start Date: Feb. 05, 2025
Study Type: INTERVENTIONAL
Condition:
Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA)
Intervention:
DRUG: nL-UBTF-001
Personalized antisense oligonucleotide
Recruitment Status: ACTIVE_NOT_RECRUITING
Sponsor: n-L...