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Expert Opinion on Pharmacotherapy Volume 13, 2012 - Issue 16
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Treatment options for lysosomal storage disorders: developing insights

Carin M van GelderErasmus MC University Medical Center, Center for Lysosomal and Metabolic Diseases, Department of Paediatrics, Dr. Molewaterplein 60, 3015 GJ, Rotterdam, The Netherlands
, MD,
Audrey AM VollebregtErasmus MC University Medical Center, Center for Lysosomal and Metabolic Diseases, Department of Paediatrics, Dr. Molewaterplein 60, 3015 GJ, Rotterdam, The Netherlands
, MD,
Iris PlugErasmus MC University Medical Center, Center for Lysosomal and Metabolic Diseases, Department of Paediatrics, Dr. Molewaterplein 60, 3015 GJ, Rotterdam, The Netherlands
, PhD,
Ans T van der PloegErasmus MC University Medical Center, Center for Lysosomal and Metabolic Disorders, Division of Metabolic Disease and Genetics, Department of Paediatrics, P.O. Box 2060, 3000 CB Rotterdam, The Netherlands+00 0 31 10 703 2076; +00 0 31 10 703 6800; [email protected]
, MD PhD &
Arnold JJ ReuserErasmus MC University Medical Center, Center for Lysosomal and Metabolic Diseases, Department of Clinical Genetics, Dr. Molewaterplein 50, 3015 GE, Rotterdam, The Netherlands
, PhD
Pages 2281-2299 | Published online: 26 Sep 2012

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Dolan Sondhi, Jonathan B Rosenberg, Benjamin G Van de Graaf, Stephen M Kaminsky & Ronald G Crystal. (2013) Advances in the treatment of neuronal ceroid lipofuscinosis. Expert Opinion on Orphan Drugs 1:12, pages 951-975.
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Articles from other publishers (18)

Giancarlo Parenti, Diego L Medina & Andrea Ballabio. (2021) The rapidly evolving view of lysosomal storage diseases. EMBO Molecular Medicine 13:2.
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Maria Teresa Valenti, Michela Serena, Luca Dalle Carbonare & Donato Zipeto. (2019) CRISPR/Cas system: An emerging technology in stem cell research. World Journal of Stem Cells 11:11, pages 937-956.
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Z. Begum Yagci, Elif Esvap, Hatice Asuman Ozkara, Kutlu O. Ulgen & Elif Ozkirimli Olmez. 2019. Molecular Chaperones in Human Disorders. Molecular Chaperones in Human Disorders 153 219 .
Ke Lei, Yanxia Zhao, Lirong Sun, Hui Liang, Ronghua Luo, Xiaojing Sun, Yanling Tao, Lijun Chen, Lingling Zhang, Aimin Li, Fu Li & Hongfang Ding. (2018) A pilot screening of high-risk Gaucher disease children using dried blood spot methods in Shandong province of China. Orphanet Journal of Rare Diseases 13:1.
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Chloe Christensen & Francis Choy. (2017) A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells. Diseases 5:1, pages 6.
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Christopher J. Folts, Nicole Scott-Hewitt, Christoph Pröschel, Margot Mayer-Pröschel & Mark Noble. (2016) Lysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular Toxicity. PLOS Biology 14:12, pages e1002583.
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Keiichi Motoyama, Yumi Hirai, Rena Nishiyama, Yuki Maeda, Taishi Higashi, Yoichi Ishitsuka, Yuki Kondo, Tetsumi Irie, Takumi Era & Hidetoshi Arima. (2015) Cholesterol lowering effects of mono-lactose-appended β-cyclodextrin in Niemann–Pick type C disease-like HepG2 cells. Beilstein Journal of Organic Chemistry 11, pages 2079-2086.
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Yuki Maeda, Keiichi Motoyama, Taishi Higashi, Yuka Horikoshi, Toru Takeo, Naomi Nakagata, Yuki Kurauchi, Hiroshi Katsuki, Yoichi Ishitsuka, Yuki Kondo, Tetsumi Irie, Hirokazu Furuya, Takumi Era & Hidetoshi Arima. (2015) Effects of cyclodextrins on GM1-gangliosides in fibroblasts from GM1-gangliosidosis patients. Journal of Pharmacy and Pharmacology 67:8, pages 1133-1142.
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Richard EgletonSteven J. Gray, R Jude Samulski & Sahana Nagabhushan Kalburgi. 2015. Advances in Non-Invasive Drug Delivery to the Brain. Advances in Non-Invasive Drug Delivery to the Brain 128 142 .
Stefanie Kung, Mark Walters, Peter Claes, Peter LeSouef, Jack Goldblatt, Andrew Martin, Shanti Balasubramaniam & Gareth Baynam. 2015. JIMD Reports, Volume 22. JIMD Reports, Volume 22 99 106 .
Seng H. Cheng. (2014) Gene therapy for the neurological manifestations in lysosomal storage disorders. Journal of Lipid Research 55:9, pages 1827-1838.
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Roberto Giugliani, Christina Lampe, Nathalie Guffon, David Ketteridge, Elisa Leão‐Teles, James E. Wraith, Simon A. Jones, Cheri Piscia‐Nichols, Ping Lin, Adrian Quartel & Paul Harmatz. (2014) Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux–Lamy syndrome)—10‐year follow‐up of patients who previously participated in an MPS VI survey study. American Journal of Medical Genetics Part A 164:8, pages 1953-1964.
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Giancarlo Parenti, Marco Moracci, Simona Fecarotta & Generoso Andria. (2014) Pharmacological chaperone therapy for lysosomal storage diseases. Future Medicinal Chemistry 6:9, pages 1031-1045.
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Antoine Joosten, Camille Decroocq, Julien de Sousa, Jérémy P. Schneider, Emile Etamé, Anne Bodlenner, Terry D. Butters & Philippe Compain. (2014) A Systematic Investigation of Iminosugar Click Clusters as Pharmacological Chaperones for the Treatment of Gaucher Disease. ChemBioChem 15:2, pages 309-319.
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Christian Grimm & Math P. Cuajungco. 2014. Pathologies of Calcium Channels. Pathologies of Calcium Channels 365 379 .
Xu He, Owen Pierce, Thomas Haselhorst, Mark von Itzstein, Daniel Kolarich, Nicolle H. Packer, Tracey M. Gloster, David J. Vocadlo, Yi Qian, Doug Brooks & Allison R. Kermode. (2013) Characterization and downstream mannose phosphorylation of human recombinant α-L-iduronidase produced in Arabidopsis complex glycan-deficient ( cgl ) seeds . Plant Biotechnology Journal 11:9, pages 1034-1043.
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G. Pierre. (2013) Neurodegenerative disorders and metabolic disease. Archives of Disease in Childhood 98:8, pages 618-624.
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Hanna Appelqvist, Petra Wäster, Katarina Kågedal & Karin Öllinger. (2013) The lysosome: from waste bag to potential therapeutic target. Journal of Molecular Cell Biology 5:4, pages 214-226.
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