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Azam Safary, Hakimeh Moghaddas-Sani, Mostafa Akbarzadeh-Khiavi, Alireza Khabbazzi, Mohammad A. Rafi & Yadollah Omidi. (2021) Enzyme replacement combinational therapy: effective treatments for mucopolysaccharidoses. Expert Opinion on Biological Therapy 21:9, pages 1181-1197.
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Shawn C. Aylward, Jonathan Pindrik, Nicolas J. Abreu, W. Bruce Cherny, Matthew O’Neal & Emily de Los Reyes. (2020) Cerliponase alfa for CLN2 disease, a promising therapy. Expert Opinion on Orphan Drugs 8:11, pages 445-454.
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Kazuo Ueda & Jiro Hokugo. (2020) Safety and efficacy of idursulfase in the treatment of mucopolysaccharidosis II (Hunter syndrome): a post-marketing study in Japan. Expert Opinion on Drug Safety 19:7, pages 891-901.
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Articles from other publishers (31)
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Joseph Muenzer, Barbara K. Burton, Hernan M. Amartino, Paul R. Harmatz, Luis González Gutiérrez-Solana, Matilde Ruiz-Garcia, Yuna Wu, David Merberg, David Alexanderian & Simon A. Jones. (2023) Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study. Orphanet Journal of Rare Diseases 18:1.
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Karen S. Yee, Costel Chirila, Eric Davenport, Deirdre Mladsi, Christine Barnett & William G. Kronenberger. (2023) A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT. Orphanet Journal of Rare Diseases 18:1.
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Mei-Yan Chan, Andrew Jack Nelson & Lock-Hock Ngu. (2023) Long-term experience with idursulfase beta (Hunterase) in two adolescent patients with MPS II: A case series. Molecular Genetics and Metabolism Reports 36, pages 100991.
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Carlos Eduardo Diaz Jacques, Gilian Guerreiro, Franciele Fatima Lopes, Carolina F. Moura de Souza, Roberto Giugliani & Carmen Regla Vargas. (2023) Alterations of Plasmatic Biomarkers of Neurodegeneration in Mucopolysaccharidosis Type II Patients Under Enzyme Replacement Therapy. Cell Biochemistry and Biophysics 81:3, pages 533-542.
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Nato D. Vashakmadze, Nataliya V. Zhurkova, Ekaterina Yu. Zakharova, Ludmila K. Mikhaylova & Marina A. Babaykina. (2023) Idursulfase Beta (Hunterase) Therapeutic Experience in a Patient with Mucopolysaccharidosis Type II. Current Pediatrics 22:4, pages 324-330.
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Tomoki Fukatsu, Hanae Morio, Tomomi Furihata & Hiroyuki Sonoda. (2023) Transferrin receptor-targeting property of pabinafusp alfa facilitates its uptake by various types of human brain-derived cells in vitro. Frontiers in Drug Delivery 3.
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Federica Conte, Juda-El Sam, Dirk J. Lefeber & Robert Passier. (2023) Metabolic Cardiomyopathies and Cardiac Defects in Inherited Disorders of Carbohydrate Metabolism: A Systematic Review. International Journal of Molecular Sciences 24:10, pages 8632.
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Margie A. Ream, Wendy K.K. Lam, Scott D. Grosse, Jelili Ojodu, Elizabeth Jones, Lisa A. Prosser, Angela M. Rosé, Anne Marie Comeau, Susan Tanksley, Cynthia M. Powell & Alex R. Kemper. (2023) Evidence and recommendation for mucopolysaccharidosis type II newborn screening in the United States. Genetics in Medicine 25:2, pages 100330.
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Zhenjie Zhang, Mingsheng Ma, Weimin Zhang, Yu Zhou, Fengxia Yao, Lisi Zhu, Min Wei & Zhengqing Qiu. (2023) Phenotypic and genetic characteristics of 130 patients with mucopolysaccharidosis type II: A single-center retrospective study in China. Frontiers in Genetics 14.
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Francesca Maccari, Laura Rigon, Veronica Mantovani, Fabio Galeotti, Marika Salvalaio, Francesca D’Avanzo, Alessandra Zanetti, Federica Capitani, Orazio Gabrielli, Rosella Tomanin & Nicola Volpi. (2022) Glycosaminoglycan signatures in body fluids of mucopolysaccharidosis type II mouse model under long-term enzyme replacement therapy. Journal of Molecular Medicine.
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Yuhua Qu, Hao Liu, Likun Wei, Shushan Nie, Wenjiao Ding, Sha Liu, Haiyan Liu & Hua Jiang. (2022) The Outcome of Allogeneic Hematopoietic Stem Cell Transplantation From Different Donors in Recipients With Mucopolysaccharidosis. Frontiers in Pediatrics 10.
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Vincenza Gragnaniello, Silvia Carraro, Laura Rubert, Daniela Gueraldi, Chiara Cazzorla, Pamela Massa, Stefania Zanconato & Alberto B. Burlina. (2022) A new strategy of desensitization in mucopolysaccharidosis type II disease treated with idursulfase therapy: A case report and review of the literature. Molecular Genetics and Metabolism Reports 31, pages 100878.
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Hideto Morimoto, Hiroki Morioka, Atsushi Imakiire, Ryuji Yamamoto, Tohru Hirato, Hiroyuki Sonoda & Kohtaro Minami. (2022) Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice. Molecular Therapy - Methods & Clinical Development 25, pages 534-544.
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Hwa Young Kim, Man Jin Kim, Moon-Woo Seong & Jung Min Ko. (2022) Skewed X-Chromosome Inactivation in a Korean Girl with Severe Mucopolysaccharidosis Type II. Annals of Laboratory Medicine 42:3, pages 373-375.
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Annie Arguello, René Meisner, Elliot R. Thomsen, Hoang N. Nguyen, Ritesh Ravi, Jeffrey Simms, Iris Lo, Jessica Speckart, Julia Holtzman, Thomas M. Gill, Darren Chan, Yuhsiang Cheng, Chi-Lu Chiu, Jason C. Dugas, Meng Fang, Isabel A. Lopez, Hilda Solanoy, Buyankhishig Tsogtbaatar, Yuda Zhu, Akhil Bhalla, Kirk R. Henne, Anastasia G. Henry, Anthony Delucchi, Simona Costanzo, Jeffrey M. Harris, Dolores Diaz, Kimberly Scearce-Levie & Pascal E. Sanchez. (2021) Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome. JCI Insight 6:19.
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Paweł Zapolnik & Antoni Pyrkosz. (2021) Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities. International Journal of Molecular Sciences 22:11, pages 5490.
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