Niemann–Pick Disease Type C
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Descrição
Niemann-Pick disease type C (NPC) is a disabling, lysosomal-storage disorder that has been diagnosed prenatally, neonatally, during childhood, and even into adulthood.

Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library

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