Clinical Research Directory

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 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 Bainbridge-Ropers Syndrome (BRPS) due to a pathogenic, de novo nonsense variant in ASXL3

Official title: An Open-Label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Bainbridge-Ropers Syndrome Due to ASXL3 Gene Variant

Key Details

Conditions

Interventions

Locations (1)