Treatment Options for Niemann-Pick Type C

Elizabeth Berry-Kravis, MD, PhD, co-director of the Molecular Diagnostics Section of the Genetic Laboratory at Rush Medical College in Chicago, describes dif

Treatment Options Australian NPC Disease Foundation Inc

Niemann-Pick disease treatment: a systematic review of clinical trials.

What is Niemann-Pick Type C Disease?

mRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts

Adult Onset Niemann-Pick type C (NPC) – NNPDF

Niemann–Pick Disease Type C

Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann–Pick Type C Using Quantitative Proteomics

Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Protocol for initiation of miglustat treatment in NP-C. Reproduced with

Niemann-Pick disease type C: Video & Anatomy

Patient with Niemann-Pick disease type C: over 20 years' follow-up

Central sleep apnea and daytime sleepiness in Niemann-Pick type C disease: a report of 2 cases