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Medical Article
Introduction Niemann Pick disease type C (NPC) is a rare, genetic, progressive neurodegenerative disorder characterized by impaired lysosomal function and accumulation of lipids due to dysfunctional NPC1 or NPC2 proteins, leading to multisystem involvement and progressive neurological decline (1). Clinical manifestations are heterogeneous and inclu

Arimoclomol Phase 2/3 Trial: Slowing Disease Progression in Niemann Pick Disease Type C (NPC)
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