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.

Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for PCARP (Posterior Column Ataxia With Retinitis Pigmentosa) Disease Due to Mutations in FLVCR1

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Antisense Oligonucleotide Treatment for PCARP Disease Due to Mutation in FLVCR1

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