Therapeutic Interventions in Niemann-Pick Disease Type C: A Systematic Review of Clinical Outcomes and Safety

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27 Oct 2026 0 comments

Background: Niemann-Pick disease type C (NPC) is a rare, progressive lysosomal storage disorder characterized by impaired intracellular lipid trafficking, leading to systemic, neurological, and psychiatric manifestations. Although supportive care was historically the main treatment approach, several disease-modifying and symptomatic therapies have recently emerged. This systematic review aimed to evaluate the efficacy and safety of current therapeutic strategies for NPC. Methods: A systematic...