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
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.
100 项与 nL-FLVC-001 相关的临床结果
100 项与 nL-FLVC-001 相关的转化医学
100 项与 nL-FLVC-001 相关的专利(医药)
100 项与 nL-FLVC-001 相关的药物交易