Baby With Niemann-Pick Disease — Marian McGlocklin Story

Niemann Pick Disease Type C - Symptoms, Causes, Treatment

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

Rare Diseases Research: Clinical Trial for Niemann-Pick Type C

Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

JCM, Free Full-Text

The Importance of Newborn Screening for Niemann-Pick Disease – NNPDF

At a glance: the largest Niemann-Pick type C1 cohort with 602

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological

Treatment outcomes following continuous miglustat therapy in

Niemann-Pick disease type C as a neurovisceral disease. Schematic