This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with dentatorubral-pallidoluysian atrophy (DRPLA) due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1
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Ataxia
Timeframe: Baseline to 24 months
Ataxia
Timeframe: Baseline to 24 months
Ataxia
Timeframe: Baseline to 24 months