Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1
Status:
ENROLLING_BY_INVITATION
Trial end date:
2028-12-31
Target enrollment:
Participant gender:
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.