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Enzyme replacement therapy

Rapid Immunochromatographic Detection of Serum Anti-α-Galactosidase A Antibodies in Fabry Patients after Enzyme Replacement Therapy

Sachie Nakano, Takahiro Tsukimura, Tadayasu Togawa, Toya Ohashi, Masahisa Kobayashi, Katsuyoshi Takayama, Yukuharu Kobayashi, Hiroshi Abiko, Masatsugu Satou, Tohru Nakahata, David G. Warnock, Hitoshi Sakuraba, Futoshi Shibasaki

published 17 Jun 2015

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Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease

Richat Abbas, Glen Park, Bharat Damle, Raul Chertkoff, Sari Alon

published 08 Jun 2015

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Effects of Enzyme Replacement Therapy Started Late in a Murine Model of Mucopolysaccharidosis Type I

Gabriela Pasqualim, Guilherme Baldo, Talita Giacomet de Carvalho, Angela Maria Vicente Tavares, Roberto Giugliani, Ursula Matte

published 03 Feb 2015

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Mosaicism of Podocyte Involvement Is Related to Podocyte Injury in Females with Fabry Disease

Michael Mauer, Emily Glynn, Einar Svarstad, Camilla Tøndel, Marie-Claire Gubler, Michael West, Alexey Sokolovskiy, Chester Whitley, Behzad Najafian

published 11 Nov 2014

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Increased Arterial Diameters in the Posterior Cerebral Circulation in Men with Fabry Disease

Nurcan Üçeyler, György A. Homola, Hans Guerrero González, Daniela Kramer, Christoph Wanner, Frank Weidemann, László Solymosi, Claudia Sommer

published 27 Jan 2014

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The Effect of Recombinant Human Iduronate-2-Sulfatase (Idursulfase) on Growth in Young Patients with Mucopolysaccharidosis Type II

Zbigniew Żuber, Agnieszka Różdżyńska-Świątkowska, Agnieszka Jurecka, Anna Tylki-Szymańska

published 13 Jan 2014

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Glucosylsphingosine Is a Highly Sensitive and Specific Biomarker for Primary Diagnostic and Follow-Up Monitoring in Gaucher Disease in a Non-Jewish, Caucasian Cohort of Gaucher Disease Patients

Arndt Rolfs, Anne-Katrin Giese, Ulrike Grittner, Daniel Mascher, Deborah Elstein, Ari Zimran, Tobias Böttcher, Jan Lukas, Rayk Hübner, Uta Gölnitz, Anja Röhle, Ales Dudesek, Wolfgang Meyer, Matthias Wittstock, Hermann Mascher

published 20 Nov 2013

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Myostatin and Insulin-Like Growth Factor I: Potential Therapeutic Biomarkers for Pompe Disease

Yin-Hsiu Chien, Der-Sheng Han, Wuh-Liang Hwu, Beth L. Thurberg, Wei-Shiung Yang

published 14 Aug 2013

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A Rapid and Sensitive Method for Measuring N-Acetylglucosaminidase Activity in Cultured Cells

Victor Mauri, Parisa Lotfi, Laura Segatori, Marco Sardiello

published 28 Jun 2013

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Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT

Suhrad G. Banugaria, Sean N. Prater, Trusha T. Patel, Stephanie M. DeArmey, Christie Milleson, Kathryn B. Sheets, Deeksha S. Bali, Catherine W. Rehder, Julian A. J. Raiman, Raymond A. Wang, Francois Labarthe, Joel Charrow, Paul Harmatz, Pranesh Chakraborty, Amy S. Rosenberg, Priya S. Kishnani

published 25 Jun 2013

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Illness Perception and Clinical Treatment Experiences in Patients with M. Maroteaux-Lamy (Mucopolysaccharidosis Type VI) and a Turkish Migration Background in Germany

Hansjörg Dilger, Linn Leissner, Lenka Bosanska, Christina Lampe, Ursula Plöckinger

published 24 Jun 2013

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Liver Fibrosis in Type I Gaucher Disease: Magnetic Resonance Imaging, Transient Elastography and Parameters of Iron Storage

Anneloes E. Bohte, Laura van Dussen, Erik M. Akkerman, Aart J. Nederveen, Ralph Sinkus, Peter L. M. Jansen, Jaap Stoker, Carla E. M. Hollak

published 15 Mar 2013

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Long-Term Effect of Antibodies against Infused Alpha-Galactosidase A in Fabry Disease on Plasma and Urinary (lyso)Gb3 Reduction and Treatment Outcome

Saskia M. Rombach, Johannes M. F. G. Aerts, Ben J. H. M. Poorthuis, Johanna E. M. Groener, Wilma Donker-Koopman, Erik Hendriks, Mina Mirzaian, Sijmen Kuiper, Frits A. Wijburg, Carla E. M. Hollak, Gabor E. Linthorst

published 19 Oct 2012

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