Niemann–Pick Disease Type C

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

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

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

Niemann-Pick Disease Type C – EffRx

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

Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium

Niemann–Pick disease type C1 (NPC1) is associated with early-onset

A Rare Case of Late Adult-Onset Niemann-Pick Disease Type C

NNPDF on X: October is Global Niemann-Pick Disease Awareness Month! For more information on Niemann-Pick Disease or to make a donation to NNPDF go to #niemannpick #ASMD #NPC #raredisease #NNPDF #NiemannPickC

Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Main therapies and point of action in Niemann type C disease.

Proposed pathological cascade in Niemann-Pick disease type C (NPC)

IJMS, Free Full-Text

Frontiers Lysosomal and Mitochondrial Liaisons in Niemann-Pick Disease

Niemann-Pick Type C « Niemann-Pick Children's Fund, Inc

Parents of kids with Niemann Pick C advocate for adrabetadex