Niemann-Pick B - RS - 5th infusion.

Pediatric hepatocellular carcinoma associated with Niemann–Pick disease type C: Case report and literature review - Hwang - 2023 - JIMD Reports - Wiley Online Library

Investigating the Mechanism of Cyclodextrins in the Treatment of Niemann‐ Pick Disease Type C Using Crosslinked 2‐Hydroxypropyl‐β‐cyclodextrin - Carradori - 2020 - Small - Wiley Online Library

Advances in therapies for neurological lysosomal storage disorders - Ellison - 2023 - Journal of Inherited Metabolic Disease - Wiley Online Library

Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann–Pick disease

Niemann–Pick disease - Wikipedia

Neurological manifestations of lysosomal disorders and emerging therapies targeting the CNS - The Lancet Child & Adolescent Health

New Treatment of Disease that Swells Organs Gives New Hope to 4-year-old Girl - International Niemann-Pick Disease Alliance (INPDA)

Bilateral Cystic Bronchiectasis as Novel Phenotype of Niemann-Pick Disease Type B Successfully Treated With Double Lung Transplantation - CHEST

Frontiers Expert opinion on patient journey, diagnosis and clinical monitoring in acid sphingomyelinase deficiency in Turkey: a pediatric metabolic disease specialist's perspective

Interstitial lung disease in lysosomal storage disorders

PDF) Intestinal Absorption of -Tocotrienol Is Mediated by Niemann-Pick C1-Like 1: In Situ Rat Intestinal Perfusion Studies

Fluorinated Chaperone−β-Cyclodextrin Formulations for β-Glucocerebrosidase Activity Enhancement in Neuronopathic Gaucher Disease

Natural history and management of liver dysfunction in lysosomal storage disorders

Sphingomyelin-induced inhibition of the plasma membrane calcium ATPase causes neurodegeneration in type A Niemann–Pick disease

WO2022026410A2 - Compositions and methods for the treatment of niemann-pick type c1 disease - Google Patents