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Expert Opinion on Orphan Drugs Volume 3, 2015 - Issue 5
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Review

Understanding and managing parkinsonism in patients with glucocerebrosidase mutations

Jenny KimNational Institutes of Health, National Human Genome Research Institute, Medical Genetics Branch, Bethesda, MD 20892-3708, USA
,
Ellen SidranskyNational Institutes of Health, National Human Genome Research Institute, Medical Genetics Branch, Building 35A, Room 1E623, Bethesda, MD 20892-3708, USA+1 301 451 0913; +1 301 402 6438; [email protected]
, MD &
Grisel LopezNational Institutes of Health, National Human Genome Research Institute, Medical Genetics Branch, Bethesda, MD 20892-3708, USA
, MD
Pages 549-562 | Published online: 23 Apr 2015

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Christian J. Hendriksz, Mathieu Anheim, Peter Bauer, Olivier Bonnot, Anupam Chakrapani, Jean-Christophe Corvol, Tom J. de Koning, Anna Degtyareva, Carlo Dionisi-Vici, Sarah Doss, Thomas Duning, Paola Giunti, Rosa Iodice, Tracy Johnston, Dierdre Kelly, Hans-Hermann Klünemann, Stefan Lorenzl, Alessandro Padovani, Miguel Pocovi, Matthis Synofzik, Alta Terblanche, Florian Then Bergh, Meral Topçu, Christine Tranchant, Mark Walterfang, Christian Velten & Stefan A. Kolb. (2017) The hidden Niemann-Pick type C patient: clinical niches for a rare inherited metabolic disease. Current Medical Research and Opinion 33:5, pages 877-890.
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