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Clinical Trials associated with nL-ATN1-002 / Active, not recruitingPhase 1/2IIT An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for ATN1 Gene Mutation
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
100 Clinical Results associated with nL-ATN1-002
100 Translational Medicine associated with nL-ATN1-002
100 Patents (Medical) associated with nL-ATN1-002
100 Deals associated with nL-ATN1-002