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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
Conditions
Interventions
nL-FLVC-001
Locations
1
United States
Childrens Hospital Colorado
Aurora, Colorado, United States
Start Date
August 23, 2023
Primary Completion Date
August 21, 2024
Completion Date
December 31, 2028
Last Updated
January 27, 2025
Lead Sponsor
University of Colorado, Denver
Data Source & Attribution
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