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Enzyme replacement therapy

Nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II

Alex J. Shamoun, Gisienne Reis, Malaica Ashley, Anatalia Labilloy, Leonardo F. Ferreira

published 26 Jun 2025

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Exploring the diagnostic potential of miRNA signatures in the Fabry disease serum: A comparative study of automated and manual sample isolations

Josephine Y. Fang, Saravanan Ayyadurai, Alyssa F. Pybus, Hiroshi Sugimoto, Mark G. Qian

published 28 Oct 2024

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Association between under-dose of enzyme replacement therapy and quality of life in adults with late-onset Pompe disease in China: A retrospective matched cohort study

Huanyu Zhang, Shanquan Chen, Richard Huan Xu, Siyue Yu, Jiazhou Yu, Dong Dong

published 17 Sep 2024

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Discovery of allosteric regulators with clinical potential to stabilize alpha-L-iduronidase in mucopolysaccharidosis type I

Elena Cubero, Ana Ruano, Aida Delgado, Xavier Barril, Sara Morales, Ana Trapero, Lorenzo Leoni, Manolo Bellotto, Roberto Maj, Beatriz Calvo-Flores Guzmán, Natalia Pérez-Carmona, Ana Maria Garcia-Collazo

published 20 May 2024

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Circulatory C-type natriuretic peptide reduces mucopolysaccharidosis-associated craniofacial hypoplasia in vivo

Marina Kashiwagi, Kazumasa Nakao, Shigeki Yamanaka, Ichiro Yamauchi, Takafumi Yamashita, Toshihito Fujii, Yohei Ueda, Mariko Yamamoto Kawai, Takuma Watanabe, Shizuko Fukuhara, Kazuhisa Bessho

published 10 Nov 2022

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Screening for Fabry Disease in patients with unexplained left ventricular hypertrophy

Chandu Sadasivan, Josie T. Y. Chow, Bun Sheng, David K. H. Chan, Yiting Fan, Paul C. L. Choi, Jeffrey K. T. Wong, Mabel M. B. Tong, Tsz-Ngai Chan, Erik Fung, Kevin K. H. Kam, Joseph Y. S. Chan, Wai-Kin Chi, D. Ian Paterson, Manohara Senaratne, Neil Brass, Gavin Y. Oudit, Alex P. W. Lee

published 28 Sep 2020

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Self-association of human beta-galactocerebrosidase: Dependence on pH, salt, and surfactant

Eunhee Lee, Nazila Salamat-Miller, Walter F. Stafford, Katherine Taylor

published 23 Dec 2019

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Japanese nationwide survey of hypophosphatasia reveals prominent differences in genetic and dental findings between odonto and non-odonto types

Rena Okawa, Kazuma Kokomoto, Taichi Kitaoka, Takuo Kubota, Atsushi Watanabe, Takeshi Taketani, Toshimi Michigami, Keiichi Ozono, Kazuhiko Nakano

published 10 Oct 2019

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Longitudinal study on ocular manifestations in a cohort of patients with Fabry disease

Langis Michaud

published 27 Jun 2019

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The ACE I/D polymorphism does not explain heterogeneity of natural course and response to enzyme replacement therapy in Pompe disease

Esther Kuperus, Jan C. van der Meijden, Stijn L. M. in ’t Groen, Marian A. Kroos, Marianne Hoogeveen-Westerveld, Dimitris Rizopoulos, Monica Yasmin Nino Martinez, Michelle E. Kruijshaar, Pieter A. van Doorn, Nadine A. M. E. van der Beek, Ans T. van der Ploeg, W. W. M. Pim Pijnappel

published 07 Dec 2018

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Oxidative stress and the altered reaction to it in Fabry disease: A possible target for cardiovascular-renal remodeling?

Verdiana Ravarotto, Gianni Carraro, Elisa Pagnin, Giovanni Bertoldi, Francesca Simioni, Giuseppe Maiolino, Matteo Martinato, Linda Landini, Paul A. Davis, Lorenzo A. Calò

published 27 Sep 2018

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Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy

Abhijit Ricky Pal, Jean Mercer, Simon A. Jones, Iain A. Bruce, Brian W. Bigger

published 18 Sep 2018

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Phenotype and biochemical heterogeneity in late onset Fabry disease defined by N215S mutation

L. Lavalle, A. S. Thomas, B. Beaton, H. Ebrahim, M. Reed, U. Ramaswami, P. Elliott, A. B. Mehta, D. A. Hughes

published 05 Apr 2018

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