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Vincenzo Di Lazzaro, Massimo Marano, Lucia Florio & Stefano De Santis. (2016) Niemann–Pick type C: focus on the adolescent/adult onset form. International Journal of Neuroscience 126:11, pages 963-971.
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Kristina L Elvidge, John Christodoulou, Michelle A Farrar, Dominic Tilden, Megan Maack, Madeline Valeri, Magda Ellis, Nicholas J C Smith, David R Thorburn, Gail Hilton, Ellie Van Velsen, Danielle Cini, Briana Davis, Richard Webster, Carolyn J Ellaway & Anita Inwood. (2023) The collective burden of childhood dementia: a scoping review. Brain 146:11, pages 4446-4455.
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Medo Gutić, Miloš N. Milosavljević & Slobodan M. Janković. (2022) Cost-effectiveness of miglustat versus symptomatic therapy of Niemann–Pick disease type C. International Journal of Clinical Pharmacy 44:6, pages 1442-1453.
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George A. Diaz, Joslyn Crowe & Justin Hopkin. (2022) Health insurance literacy and health services access barriers in Niemann–Pick disease: the patient and caregiver voice. Orphanet Journal of Rare Diseases 17:1.
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Elias Seidl, Nicolaus Schwerk, Julia Carlens, Martin Wetzke, Steve Cunningham, Nagehan Emiralioğlu, Nural Kiper, Joanna Lange, Katarzyna Krenke, Nicola Ullmann, Dora Krikovszky, Phillen Maqhuzu, Charlotte A Griese, Larissa Schwarzkopf & Matthias Griese. (2022) Healthcare resource utilisation and medical costs for children with interstitial lung diseases (chILD) in Europe. Thorax 77:8, pages 781-789.
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Lidia García-Pérez, Renata Linertová, Cristina Valcárcel-Nazco, Manuel Posada, Inigo Gorostiza & Pedro Serrano-Aguilar. (2021) Cost-of-illness studies in rare diseases: a scoping review. Orphanet Journal of Rare Diseases 16:1.
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Joanna Leśniowska. (2020) Economic Burden of Rare Diseases With Common Diseases as a Comorbidity in Poland. Problemy Zarządzania - Management Issues 2/2020:88, pages 103-120.
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Yllka Kodra, Marianna Cavazza, Marta de Santis, Andrea Guala, Maria-Elena Liverani, Patrizio Armeni, Maura Masini & Domenica Taruscio. (2020) Social Economic Costs, Health-Related Quality of Life and Disability in Patients with Cri Du Chat Syndrome. International Journal of Environmental Research and Public Health 17:16, pages 5951.
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Sana Eljamel, Wrik Ghosh, Sachin De Stone, Annabel Griffiths, Timothy Barrett & Richard Thompson. (2019) A cost of illness study evaluating the burden of Wolfram syndrome in the United Kingdom. Orphanet Journal of Rare Diseases 14:1.
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Anita W. M. Suijkerbuijk, Corien M. Swaan, Marie-Josee J. Mangen, Johan J. Polder, Aura Timen & Wilhelmina L. M. Ruijs. (2017) Ebola in the Netherlands, 2014–2015: costs of preparedness and response. The European Journal of Health Economics 19:7, pages 935-943.
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Giulia Polo, Alessandro Burlina, Francesca Furlan, Thilini Kolamunnage, Mara Cananzi, Laura Giordano, Martina Zaninotto, Mario Plebani & Alberto Burlina. (2016) High level of oxysterols in neonatal cholestasis: a pitfall in analysis of biochemical markers for Niemann-Pick type C disease. Clinical Chemistry and Laboratory Medicine (CCLM) 54:7, pages 1221-1229.
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Georgi Iskrov, Itziar Astigarraga, Rumen Stefanov, Julio López-Bastida, Renata Linertová, Juan Oliva-Moreno, Pedro Serrano-Aguilar, Manuel Posada-de-la-Paz, Arrigo Schieppati, Domenica Taruscio, Márta Péntek, Johann Matthias Graf von der Schulenburg, Panos Kanavos, Karine Chevreul, Ulf Persson & Giovanni Fattore. (2016) Social/economic costs and health-related quality of life in patients with histiocytosis in Europe. The European Journal of Health Economics 17:S1, pages 67-78.
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Jackie Imrie, Lesley Heptinstall, Stephen Knight & Kate Strong. (2015) Observational cohort study of the natural history of Niemann-Pick disease type C in the UK: a 5-year update from the UK clinical database. BMC Neurology 15:1.
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Aris Angelis, David Tordrup & Panos Kanavos. (2015) Socio-economic burden of rare diseases: A systematic review of cost of illness evidence. Health Policy 119:7, pages 964-979.
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Clara D.M. van Karnebeek, Tima Mohammadi, Nicole Tsao, Graham Sinclair, Sandra Sirrs, Sylvia Stockler & Carlo Marra. (2015) Health economic evaluation of plasma oxysterol screening in the diagnosis of Niemann–Pick Type C disease among intellectually disabled using discrete event simulation. Molecular Genetics and Metabolism 114:2, pages 226-232.
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Marie T Vanier. (2010) Niemann-Pick disease type C. Orphanet Journal of Rare Diseases 5:1.
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