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

U.S., Sept. 30 -- ClinicalTrials.gov registry received information related to the study (NCT07197294) titled 'Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)' on July 23.

Brief Summary: 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

Study Start Date: Feb. 24

Study Type: INTERVENTIONAL

Condition: Neurodevelopmental Disorder With or Without Variable Brain Abnormalities

Intervention: DRUG: nL-MAPK8-001

Persona...