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

From genes to hope

Liposome-targeted recombinant human acid sphingomyelinase: Production, formulation, and in vitro evaluation - ScienceDirect

Speaker Series - Niemann-Pick Disease Type C

IJMS, Free Full-Text

Niemann-Pick Disease Type C

Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrin

Niemann-Pick disease type C Alzheimer Society of Canada

Niemann-Pick type C disease – the tip of the iceberg? A review of neuropsychiatric presentation, diagnosis and treatment, BJPsych Bulletin

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

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

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