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Shilpa A Patil & Gustavo HB Maegawa. (2013) Developing therapeutic approaches for metachromatic leukodystrophy. Drug Design, Development and Therapy 7, pages 729-745.
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Read now
Roberto Giugliani, Andressa Federhen, André Anjos Silva, Camila Matzenbacher Bittar, Carolina Fischinger Moura de Souza, Cristina Brinckmann Oliveira Netto, Fabiana Quoos Mayer, Guilherme Baldo & Ursula Matte. (2012) Emerging treatment options for the mucopolysaccharidoses. Research and Reports in Endocrine Disorders 2, pages 53-64.
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Angela Gritti. (2011) Gene therapy for lysosomal storage disorders. Expert Opinion on Biological Therapy 11:9, pages 1153-1167.
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Articles from other publishers (28)
S. Ellison, H. Parker & B. Bigger. (2023) Advances in therapies for neurological lysosomal storage disorders. Journal of Inherited Metabolic Disease 46:5, pages 874-905.
Crossref
Crossref
Małgorzata Skweres-Kuchta, Iwona Czerska & Elżbieta Szaruga. (2023) Literature Review on Health Emigration in Rare Diseases—A Machine Learning Perspective. International Journal of Environmental Research and Public Health 20:3, pages 2483.
Crossref
Crossref
Claudia L. CravenPaul Gissen, Rebecca Bower, Laura Lee, Kristian Aquilina & Dominic N. P. Thompson. (2022) A survival analysis of ventricular access devices for delivery of cerliponase alfa. Journal of Neurosurgery: Pediatrics 29:1, pages 115-121.
Crossref
Crossref
Serena Ricci & Pietro Cacialli. (2021) Stem Cell Research Tools in Human Metabolic Disorders: An Overview. Cells 10:10, pages 2681.
Crossref
Crossref
Saumya Pandey & Afroz Alam. 2020. Advanced Pharmacological Uses of Medicinal Plants and Natural Products. Advanced Pharmacological Uses of Medicinal Plants and Natural Products
269
284
.
Shifaza Mohamed, Qi Qi He, Arti A. Singh & Vito Ferro. 2020.
71
117
.
Marta Moskot, Joanna Jakóbkiewicz-Banecka, Anna Kloska, Ewa Piotrowska, Magdalena Narajczyk & Magdalena Gabig-Cimińska. (2019) The Role of Dimethyl Sulfoxide (DMSO) in Gene Expression Modulation and Glycosaminoglycan Metabolism in Lysosomal Storage Disorders on an Example of Mucopolysaccharidosis. International Journal of Molecular Sciences 20:2, pages 304.
Crossref
Crossref
Daniela Trono. 2019. Advances in Enzyme Technology. Advances in Enzyme Technology
349
387
.
Fedah E. Mohamed, Lihadh Al-Gazali, Fatma Al-Jasmi & Bassam R. Ali. (2017) Pharmaceutical Chaperones and Proteostasis Regulators in the Therapy of Lysosomal Storage Disorders: Current Perspective and Future Promises. Frontiers in Pharmacology 8.
Crossref
Crossref
Marino Convertino, Jhuma Das & Nikolay V. Dokholyan. (2016) Pharmacological Chaperones: Design and Development of New Therapeutic Strategies for the Treatment of Conformational Diseases. ACS Chemical Biology 11:6, pages 1471-1489.
Crossref
Crossref
Maria Francisca Coutinho & Sandra Alves. (2016) From rare to common and back again: 60years of lysosomal dysfunction. Molecular Genetics and Metabolism 117:2, pages 53-65.
Crossref
Crossref
Ralf Reski, Juliana Parsons & Eva L. Decker. (2015) Moss‐made pharmaceuticals: from bench to bedside. Plant Biotechnology Journal 13:8, pages 1191-1198.
Crossref
Crossref
Camilla Parmeggiani, Serena Catarzi, Camilla Matassini, Giampiero D'Adamio, Amelia Morrone, Andrea Goti, Paolo Paoli & Francesca Cardona. (2015) Human Acid β-Glucosidase Inhibition by Carbohydrate Derived Iminosugars: Towards New Pharmacological Chaperones for Gaucher Disease. ChemBioChem 16:14, pages 2054-2064.
Crossref
Crossref
Giancarlo Parenti, Generoso Andria & Kenneth J Valenzano. (2015) Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders. Molecular Therapy 23:7, pages 1138-1148.
Crossref
Crossref
Maria Francisca Coutinho, Liliana Matos & Sandra Alves. (2015) From bedside to cell biology: A century of history on lysosomal dysfunction. Gene 555:1, pages 50-58.
Crossref
Crossref
Naveen Kumar Chennamaneni, Arun Babu Kumar, Mariana Barcenas, Zdeněk Spáčil, C. Ronald Scott, František Tureček & Michael H. Gelb. (2014) Improved Reagents for Newborn Screening of Mucopolysaccharidosis Types I, II, and VI by Tandem Mass Spectrometry. Analytical Chemistry 86:9, pages 4508-4514.
Crossref
Crossref
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.
Crossref
Crossref
Miao Xu, Ke Liu, Manju Swaroop, Wei Sun, Seameen J. Dehdashti, John C. McKew & Wei Zheng. (2014) A Phenotypic Compound Screening Assay for Lysosomal Storage Diseases. SLAS Discovery 19:1, pages 168-175.
Crossref
Crossref
M. Beck. 2014. Angeborene Stoffwechselkrankheiten bei Erwachsenen. Angeborene Stoffwechselkrankheiten bei Erwachsenen
291
303
.
D. V. Titov, M. L. Gening, Yu. E. Tsvetkov & N. E. Nifantiev. (2013) Conjugates of cyclooligosaccharide scaffolds and carbohydrate ligands: Methods for synthesis and the interaction with lectins. Russian Journal of Bioorganic Chemistry 39:5, pages 451-487.
Crossref
Crossref
Robert E. Boyd, Gary Lee, Philip Rybczynski, Elfrida R. Benjamin, Richie Khanna, Brandon A. Wustman & Kenneth J. Valenzano. (2013) Pharmacological Chaperones as Therapeutics for Lysosomal Storage Diseases. Journal of Medicinal Chemistry 56:7, pages 2705-2725.
Crossref
Crossref
Camille Decroocq, David Rodríguez-Lucena, Kyoko Ikeda, Naoki Asano & Philippe Compain. (2012) Cyclodextrin-Based Iminosugar Click Clusters: The First Examples of Multivalent Pharmacological Chaperones for the Treatment of Lysosomal Storage Disorders. ChemBioChem 13:5, pages 661-664.
Crossref
Crossref
GUSTAVO MAEGAWA. (2012) GM2 gangliosidosis: the prototype of lysosomal storage disorders. Developmental Medicine & Child Neurology 54:2, pages 104-105.
Crossref
Crossref
Haifeng Geng, Grace Whiteley, Jameson Ribbens, Wei Zheng, Noel Southall, Xin Hu, Juan J. Marugan, Marc Ferrer & Gustavo H. B. Maegawa. (2011) Novel Patient Cell-Based HTS Assay for Identification of Small Molecules for a Lysosomal Storage Disease. PLoS ONE 6:12, pages e29504.
Crossref
Crossref
. (2011) Current World Literature. Current Opinion in Pediatrics 23:6, pages 700-707.
Crossref
Crossref
Wouter Meersseman, Patrick Verschueren, Thomas Tousseyn, Rita De Vos & David Cassiman. (2011) PAS-positive macrophages—not always infection. The Lancet 377:9780, pages 1890.
Crossref
Crossref
Ana Trapero, Ignacio Alfonso, Terry D. Butters & Amadeu Llebaria. (2011) Polyhydroxylated Bicyclic Isoureas and Guanidines Are Potent Glucocerebrosidase Inhibitors and Nanomolar Enzyme Activity Enhancers in Gaucher Cells. Journal of the American Chemical Society 133:14, pages 5474-5484.
Crossref
Crossref
Mirella Filocamo, David N Cooper & Maja Di Rocco. 2005. Encyclopedia of Life Sciences. Encyclopedia of Life Sciences.