DHDDS-related juvenile parkinsonism is caused by impaired lipid metabolism, glycosylation, and mitochondrial dysfunction, which can be rescued by NAD⁺ treatment.
This study demonstrates that DHDDS-related juvenile parkinsonism arises from impaired lipid metabolism, glycosylation defects, and mitochondrial dysfunction in patient-derived brain organoids, and that these pathological features, along with associated clinical symptoms, can be effectively rescued by NAD⁺ precursor (NMN) treatment.