Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS
Status:
ACTIVE_NOT_RECRUITING
Trial end date:
2026-04-01
Target enrollment:
Participant gender:
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