Clinical Trial: Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

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...