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Ethical Issues Related to the Access to Orphan Drugs in Brazil: The Case of Mucopolysaccharidosis Type I Boy, Raquel; Schwartz, Ida V D; Krug, Bárbara C; Santana-da-Silva, Luiz C; Steiner, Carlos E; Acosta, Angelina X; Ribeiro, Erlane M; Galera, Marcial F; Leivas, Paulo G C; Braz, Marlene (2011-04)Mucopolysaccharidosis type I (MPS I) is a rare lysosomal storage disorder treated with bone marrow transplantation or enzyme replacement therapy with laronidase, a high-cost orphan drug. Laronidase was approved by the US ...