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Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS
Sponsor: n-Lorem Foundation
Summary
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10
Official title: An Open-Label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10
Key Details
Gender
All
Age Range
63 Years - 63 Years
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2024-06-24
Completion Date
2026-06
Last Updated
2025-07-25
Healthy Volunteers
No
Conditions
Interventions
nL-CHCHD-001
Personalized Antisense Oligonucleotide
Locations (1)
Columbia University, Irving Medical Center
New York, New York, United States