1. Enzyme replacement therapy with galsulfase in 34 children younger than five years of age with MPS VI
- Author
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Ana Carolina de Paula, Liane de Rosso Giuliani, Maria do Carmo S. Rodrigues, Francisca C. Santos, Angelina Xavier Acosta, Renata C.F. Bonatti, Bethania F.R. Ribeiro, Laercio Cardoso, Marcelo Kerstenestzy, Carlos Antônio Bruno da Silva, Gervina Brady Moreira Holanda, Chong Ae Kim, Maria Ione F. Costa, Tatiana S. P. C. Magalhães, Jordão Correa Neto, Durval Batista Palhares, Juan C. Llerena, Carolina Sanchez Aranda, Dafne Dain Gandelman Horovitz, Ana Maria Martins, Mara Albonei Dudeque Pianovski, Erlane Marques Ribeiro, Fiocruz MS, Universidade Federal da Bahia (UFBA), Hosp Albert Sabin, Universidade Federal de Mato Grosso do Sul (UFMS), Universidade de São Paulo (USP), Hosp Barao de Lucena, Univ Fed Parana, Ctr Reabilitacao Infantil, Hosp Univ Maranhao, Universidade Federal de São Paulo (UNIFESP), Univ Fed Rio Grande do Norte, Univ Fortaleza, Univ Fed Triangulo Mineiro, Hosp Clin Acre, and Univ Fed Espirito Santo
- Subjects
Male ,medicine.medical_specialty ,Lysosomal storage disorder ,N-Acetylgalactosamine-4-Sulfatase ,Endocrinology, Diabetes and Metabolism ,MPS VI ,Mucopolysaccharidosis type VI ,Disease ,Biochemistry ,Galsulfase ,Endocrinology ,Internal medicine ,Lysosomal storage disease ,medicine ,Genetics ,Humans ,Enzyme Replacement Therapy ,Adverse effect ,Molecular Biology ,Pediatric ,Mucopolysaccharidosis VI ,business.industry ,Medical record ,Infant, Newborn ,Infant ,Sleep apnea ,Enzyme replacement therapy ,medicine.disease ,Recombinant Proteins ,Surgery ,Child, Preschool ,Female ,business ,Follow-Up Studies - Abstract
BioMarin Pharmaceutical Inc. Shire Genzyme BioMarin Background: Mucopolysaccharidosis type VI (MPS VI) is a progressive, chronic and multisystem lysosomal storage disease with a wide disease spectrum. Clinical and biochemical improvements have been reported for MPS VI patients on enzyme replacement therapy (ERT) with rhASB (recombinant human arylsulfatase B; galsulfase, Naglazyme (R), BioMarin Pharmaceutical Inc.), making early diagnosis and intervention imperative for optimal patient outcomes. Few studies have included children younger than five years of age. This report describes 34 MPS VI patients that started treatment with galsulfase before five years of age.Methods: Data from patients who initiated treatment at
- Published
- 2013
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