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Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation
Sponsor: n-Lorem Foundation
Summary
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
Official title: An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Dentatorubral-pallidoluysian Atrophy (DRPLA) Due to ATN1 Mutation
Key Details
Gender
MALE
Age Range
22 Years - Any
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2025-11-06
Completion Date
2027-11
Last Updated
2026-04-07
Healthy Volunteers
No
Conditions
Interventions
nL-ATN1-001
Personalized Antisense Oligonucleotide
Locations (1)
Dell Children's
Austin, Texas, United States