Niemann–Pick type C disease: cellular pathology and

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Therapeutic intervention for Niemann–Pick type C disease must target

A Collection of Surgical Pathology Images

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial - The Lancet

NPC1-dependent alterations in KV2.1–CaV1.2 nanodomains drive neuronal death in models of Niemann-Pick Type C disease

Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016

Niemann-Pick Diseases - The Medical Biochemistry Page

Biophysical impact of sphingosine and other abnormal lipid accumulation in Niemann-Pick disease type C cell models - ScienceDirect

At a glance: the largest Niemann-Pick type C1 cohort with 602 patients diagnosed over 15 years

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