Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
n-Lorem Foundation
With
Thomas Jefferson University
This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP
Age
Any age
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Informed consent/assent provided by the participant (when appropriate), and/or participants parent(s) or legally authorized representative(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
You may not be if
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
Clareo Health | Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS
