Therapeutic intervention for Niemann–Pick type C disease must target

Parents of kids with Niemann Pick C advocate for adrabetadex

Potential pharmacological strategies targeting the Niemann-Pick C1 receptor and Ebola virus glycoprotein interaction - ScienceDirect

Niemann-Pick Disease Type C (NP-C) Gene Therapy Activation Meeting (GAM) - International Niemann-Pick Disease Alliance (INPDA)

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

Effect of acid sphingomyelinase deficiency in type A Niemann-Pick disease on the transport of therapeutic nanocarriers across the blood–brain barrier

Niemann–Pick Disease Type C

Niemann-Pick type C: contemporary diagnosis and treatment of a classical disorder

Therapeutic intervention for Niemann–Pick type C disease must target

Combination therapy a potential strategy for treating Niemann-Pick disease

Clinical, ocular motor, and imaging profile of Niemann-Pick type C heterozygosity

Experimental treatment for Niemann-Pick disease type C1 appears safe, effective

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

Immune dysfunction in Niemann‐Pick disease type C - Platt - 2016 - Journal of Neurochemistry - Wiley Online Library