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.
Inclusion Criteria:
Exclusion Criteria:
St Louis, Missouri 63110, United States
Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS
Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS
Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS
Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS
Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)
Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS
Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation
Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)