Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

An Open-Label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for individual participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10

Inclusion Criteria:

• Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representatives(s)
• Ability to travel to the study site and adhere to study related follow-up examinations and/or procedures and provide access to participant's medical records
• Genetically confirmed neurological disorder

Exclusion Criteria:

• Participant has any condition that in the opinion of the Site Investigator would ultimately prevent the completion of study procedures
• Use of an investigational medication within less than 5 half-lives of the drug at enrollment