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
Dell Children's Medical Center of Central Texas
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 DRPLA due to a heterozygous pathogenic CAG trinucleotide expansion in ATN1
Age
22–any
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
Informed consent/assent provided by the participant (when appropriate), and/or participant's 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 Dentatorubral-pallidoluysian atrophy (DRPLA) due to ATN1 mutation
You may not be if
Use of investigational medication within 5 half-lives of the drug at enrolment
Participant has any condition that in the opinion of the Site Investigator would ultimately prevent the completion of study procedures
Clareo Health | Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation
