Clinical Research Directory

Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

Sponsor: n-Lorem Foundation

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

Official title: An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA) Caused by UBTF Gene Mutation

Key Details

Conditions

Interventions

Locations (1)