Navegação Produção Científica por Autor "Quartel, Adrian"
Resultados 1-2 de 2
-
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 ... -
Long-term galsufase treatment associated with improved survival of patients with mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome) : 15-year follow-up from the survey study
Quartel, Adrian; Harmatz, Paul; Lampe, Christina; Guffon, Nathalie; Ketteridge, David; Teles, Elisa Leão; Jones, Simon A.; Giugliani, Roberto (2018) [Artigo de periódico]Mucopolysaccharidosis VI (MPS VI) is a progressive lysosomal storage disorder with multiorgan and multisystemic pathology. Currently, galsulfase enzyme replacement therapy (ERT) is the only approved treatment for MPS VI. ...