Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with Neurodevelopmental Disorder with or without Brain Abnormalities (NEDBA) due to a heterozygous pathogenic missense mutation in MAPK8IP3

NCT Number	NCT07197294
Lead Sponsor	n-Lorem Foundation Collaborators: Columbia University
Conditions	Neurodevelopmental Disorder With or Without Variable Brain Abnormalities
Enrollment	1 participants
Start Date	2025-02-24
Primary Completion	2027-02 (estimated)
Study Completion	2027-02 (estimated)
Updated on ClinicalTrials.gov	2025-09-29