Development of a Therapy for the Treatment of Zellweger Spectrum Disorder

This technology includes a method for selecting a therapeutic effective amount of one of two compounds (including naltriben and naltrin) for the treatment of Zellweger Spectrum Disorder (ZSD), or any disease associated with peroxisome dysfunction. The compounds were identified using a cell-image based high-content screening (HCS) assay to identify small molecules that enhance peroxisome assembly in immortalized skin fibroblasts obtained from a ZSD patient.