/ Active, not recruiting临床1/2期IIT 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 项与 nL-ATN1-002 相关的临床结果
100 项与 nL-ATN1-002 相关的转化医学
100 项与 nL-ATN1-002 相关的专利(医药)
100 项与 nL-ATN1-002 相关的药物交易