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Enzyme replacement therapy for Mucopolysaccharidosis Type I among patients foll...

Dornelles,Alícia Dorneles; Pinto,Louise Lapagesse de Camargo; Paula,Ana Carolina de; Steiner,Carlos Eduardo; Lourenço,Charles Marques; Kim,Chong Ae

Mucopolysaccharidosis type I (MPS I) is a rare lysosomal disorder caused by deficiency of alph-L-iduronidase. Few clinical trials have assessed the effect of enzyme replacement therapy (ERT) for this condition. We conducted an exploratory, open-label, non-randomized, multicenter cohort study of patients with MPS I. Data were collected from questionnaires completed by attending physicians at the time of diagnosi...

Date: 2014   |   Origin: Oasisbr
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