Navegação por Autor "Parini, Rossella"
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Clinical outcomes in a subpopulation of adults with Morquio A syndrome : results from a long-term extension study of elosulfase alfa
Hughes, Derralynn A.; Giugliani, Roberto; Guffon, Nathalie; Jones, Simon A.; Mengel, Karl Eugen; Parini, Rossella; Matousek, R.; Hawley, Sara M.; Quartel, Adrian (2017) [Artigo de periódico]Background: This post hoc subanalysis examined outcomes in adult patients with Morquio A (mucopolysaccharidosis IVA) who received enzyme replacement therapy (ERT) with elosulfase alfa over a 120-weeks period. Patients ≥18 ...