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European Journal of Immunology|May 16, 2012
Invariant natural killer T cells are not affected by lysosomal storage in patients with Niemann-Pick disease type CAnneliese O Speak, Nicholas Platt, Mariolina Salio, et al.Molecular Genetics and Metabolism Reports|June 16, 2025
Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity ScaleEugen Mengel, Marc C Patterson, Rosalia M Da Riol, et al.Blood|November 16, 2013
Altered distribution and function of natural killer cells in murine and human Niemann-Pick disease type C1Anneliese O Speak, Danielle Te Vruchte, Lianne C Davis, et al.Communications Biology|August 12, 2022
Modeling SARS-CoV-2 and influenza infections and antiviral treatments in human lung epithelial tissue equivalentsHoda Zarkoob, Anna Allué-Guardia, Yu-Chi Chen, et al.Biochimica Et Biophysica Acta. Molecular and Cell Biology of Lipids|May 4, 2019
2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 diseaseJessica Davidson, Elizabeth Molitor, Samantha Moores, et al.Current Topics in Medicinal Chemistry|November 29, 2013
Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 diseaseElizabeth A Ottinger, Mark L Kao, Nuria Carrillo-Carrasco, et al.Lancet (London, England)|August 15, 2017
Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trialDaniel S Ory, Elizabeth A Ottinger, Nicole Yanjanin Farhat, et al.Journal of Inherited Metabolic Disease|April 26, 2026
2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type CTarekegn Hiwot, Forbes D Porter, Tatiana Bremova-Ertl, et al.American Journal on Intellectual and Developmental Disabilities|November 19, 2020
Person Ability Scores as an Alternative to Norm-Referenced Scores as Outcome Measures in Studies of Neurodevelopmental DisordersCristan A Farmer, Aaron J Kaat, Audrey Thurm, et al.The Journal of Clinical Investigation|February 4, 2014
Relative acidic compartment volume as a lysosomal storage disorder-associated biomarkerDanielle te Vruchte, Anneliese O Speak, Kerri L Wallom, et al.Pageof 20