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  1. Article

    Open Access

    Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis

    Niemann-Pick Disease Type C (NPC) is an inherited, often fatal neurovisceral lysosomal storage disease characterized by cholesterol accumulation in every cell with few known treatments. Defects in cholesterol ...

    Caroline Hastings, Camilo Vieira, Benny Liu… in Orphanet Journal of Rare Diseases (2019)

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    Article

    Amyloid-β metabolism in Niemann-Pick C disease models and patients

    Niemann-Pick type C (NPC) is a progressive neurodegenerative lysosomal disease with altered cellular lipid trafficking. The metabolism of amyloid-β (Aβ) - previously mainly studied in Alzheimer’s disease - has...

    Niklas Mattsson, Maria Olsson, Mikael K. Gustavsson… in Metabolic Brain Disease (2012)

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    Article

    Isolated Extramedullary Relapse in Childhood Acute Lymphocytic Leukemia

    Although the vast majority of children with acute lymphocytic leukemia attain remission with modern therapies, an unacceptably high number will suffer a disease relapse. Both the duration of remission and the ...

    James E. Jacobs, Caroline Hastings in Current Hematologic Malignancy Reports (2010)