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Expert Opinion on Emerging Drugs Volume 21, 2016 - Issue 1
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Emerging drugs for the treatment of mucopolysaccharidoses

Roberto GiuglianiMedical Genetics Service and Clinical Research Group in Medical Genetics, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil;Department of Genetics, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;Post-Graduate Program in Child and Adolescent Health, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;Post-Graduate Program in Genetics and Molecular Biology, Universidade Federal do Rio Grande do Sul, Porto Alegre, BrazilCorrespondence[email protected]
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Andressa FederhenMedical Genetics Service and Clinical Research Group in Medical Genetics, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil;Post-Graduate Program in Child and Adolescent Health, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil
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Filippo VairoMedical Genetics Service and Clinical Research Group in Medical Genetics, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil
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Cláudia VanzellaMedical Genetics Service and Clinical Research Group in Medical Genetics, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil;Post-Graduate Program in Biological Sciences: Biochemistry, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil
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Gabriela PasqualimMedical Genetics Service and Clinical Research Group in Medical Genetics, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil;Department of Genetics, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil
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Letícia Machado Rosa da SilvaMedical Genetics Service and Clinical Research Group in Medical Genetics, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil
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Luciana GiuglianiMedical Genetics Service and Clinical Research Group in Medical Genetics, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil
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Ana Paula Kurz de BoerMedical Genetics Service and Clinical Research Group in Medical Genetics, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil
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Carolina Fishinger Moura de SouzaMedical Genetics Service and Clinical Research Group in Medical Genetics, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil
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Ursula MatteDepartment of Genetics, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;Post-Graduate Program in Genetics and Molecular Biology, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;Gene Therapy Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil
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Guilherme BaldoPost-Graduate Program in Genetics and Molecular Biology, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;Gene Therapy Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil;Department of Physiology, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil
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Pages 9-26 | Received 03 Apr 2015, Accepted 19 Nov 2015, Published online: 09 Jan 2016

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Alberto B. Burlina & Vincenza Gragnaniello. (2022) Newborn screening of mucopolysaccharidosis type I. Critical Reviews in Clinical Laboratory Sciences 59:4, pages 257-277.
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Roberto Giugliani, Amauri Dalla Corte, Fabiano Poswar, Cláudia Vanzella, Dafne Horovitz, Mariluce Riegel, Guilherme Baldo & Filippo Vairo. (2018) Intrathecal/Intracerebroventricular enzyme replacement therapy for the mucopolysaccharidoses: efficacy, safety, and prospects. Expert Opinion on Orphan Drugs 6:7, pages 403-411.
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Edward H. Schuchman, John Mitchell & Alex Solyom. (2017) Morbidity and mortality associated with Farber disease and prospects for therapy. Expert Opinion on Orphan Drugs 5:9, pages 717-726.
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Roselena S. Schuh, Guilherme Baldo & Helder F. Teixeira. (2016) Nanotechnology applied to treatment of mucopolysaccharidoses. Expert Opinion on Drug Delivery 13:12, pages 1709-1718.
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