U.S., July 25 -- ClinicalTrials.gov registry received information related to the study (NCT07084311) titled 'Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation' on May 08.
Brief 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
Study Start Date: Oct. 24, 2024
Study Type: INTERVENTIONAL
Condition:
Dentatorubral-Pallidoluysian Atrophy
Intervention:
DRUG: nL-ATN1-002
Personalized antisense oligonucleotide
Recruitment Status: ACTIVE_NOT_RECRUITING
Sponsor: n-Lorem Foundation
Published by HT Digi...