Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

Summary

The goal of this clinical trial is to evaluate a specific antisense oligonucleotide medication in one patient with posterior column ataxia with retinitis pigmentosa. The main question it aims to answer is: what is the safety and tolerability of this medication in a single participant.

Timeline

Start

2023-08-23

Primary completion

2024-08-21

Completion

2028-12-31

Drugs

Indications