Christian J. Hendriksz

Recommendations for the diagnosis and management of Niemann–Pick disease type C: An update

Marc C. Patterson, Christian J. Hendriksz, Mark Walterfang et al. · Molecular Genetics and Metabolism · 2012 · 478 citations

Medicine, Internal Medicine, Pathology +7

Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document

Marieke Biegstraaten, Reynir Arngrı́msson, Frédéric Barbey et al. · Orphanet Journal of Rare Diseases · 2015 · 345 citations · Full text

Life Sciences, Internal Medicine, Pharmacotherapy +13

Phenotypic spectrum of neurodegeneration associated with mutations in the <i>PLA2G6</i> gene (PLAN)

Manju A. Kurian, Neil V. Morgan, Lesley MacPherson et al. · Neurology · 2008 · 240 citations

Life Sciences, Genetics, Neurology +13

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo‐controlled study

Christian J. Hendriksz, Barbara K. Burton, Thomas R. Fleming et al. · Journal of Inherited Metabolic Disease · 2014 · 211 citations · Full text

Life Sciences, Pharmacology, Pharmacotherapy +12

Mortality and cause of death in mucopolysaccharidosis type II—a historical review based on data from the Hunter Outcome Survey (HOS)

Simon Jones, Zsuzsanna Almássy, Michael Beck et al. · Journal of Inherited Metabolic Disease · 2009 · 181 citations

Medicine, Pathology, Clinical Epidemiology +5