Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

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

Timeline

Start

2024-04-16

Primary completion

2027-04

Completion

2027-04

Drugs

Indications