Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016

Apostolos PAPANDREOU, Great Ormond Street Hospital for Children NHS Foundation Trust, London, GOSH, Department of Neurology

PDF) 2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1

PDF) A combination of 7-ketocholesterol, lysosphingomyelin and bile acid-408 to diagnose Niemann-Pick disease type C using LC-MS/MS

Therapeutic Strategies for Gaucher Disease: Miglustat (NB-DNJ) as a Pharmacological Chaperone for Glucocerebrosidase and the Different Thermostability of Velaglucerase Alfa and Imiglucerase

PDF) Diagnostic workup and management of patients with suspected Niemann-Pick type C disease

Mean Urinary Levels of Biomarkers in DEAEH Treatment Groups DEAEH Dose

PDF) Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1

Peter CLAYTON, University College London, London, UCL, Institute of Child Health

3 H-cholesterol efflux to Apo A-I in cultured skin fibroblasts. Cells

PDF) Diagnostic workup and management of patients with suspected Niemann-Pick type C disease

Stephanie GRÜNEWALD, Great Ormond Street Hospital for Children NHS Foundation Trust, London, GOSH, Biomedical Research Centre (BRC)

PDF) Diagnostic workup and management of patients with suspected Niemann-Pick type C disease

Mean Urinary Levels of Biomarkers in DEAEH Treatment Groups DEAEH Dose

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