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Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS
Sponsor: n-Lorem Foundation
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.
Official title: An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
6
Start Date
2026-02-19
Completion Date
2028-02
Last Updated
2026-08-03
Healthy Volunteers
No
Conditions
Interventions
nL-TARDB-006
Personalized antisense oligonucleotide
Locations (2)
Washington University
St Louis, Missouri, United States
Columbia University, Irving Medical Center
New York, New York, United States