Niemann-Pick Disease

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Emerging Therapies for Niemann-Pick Disease Type C Treatment: The Road to Progress

Niemann-Pick disease type C (NPC) is a rare genetic disorder caused by mutations in cholesterol trafficking proteins. There are two main types, with Type C1 being the most prevalent, accounting for around 95% of cases. While specific epidemiological data on NPC is limited, estimates suggest a prevalence of approxim...

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