Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome

OBJECTIVE: To present long-term respiratory function outcomes from an open-label, multi-center, phase 3 extension study (MOR-005) of elosulfase alfa enzyme replacement therapy (ERT) in patients with Morquio A syndrome. METHODS: In part 1 of MOR-005, patients initially randomized to ERT in the 24-wee...

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Publicado en:J Inherit Metab Dis
Main Authors: Hendriksz, Christian J., Berger, Kenneth I., Parini, Rossella, AlSayed, Moeenaldeen D., Raiman, Julian, Giugliani, Roberto, Mitchell, John J., Burton, Barbara K., Guelbert, Norberto, Stewart, Fiona, Hughes, Derralynn A., Matousek, Robert, Jurecki, Elaina, Decker, Celeste, Harmatz, Paul R.
Formato: Artigo
Idioma:Inglês
Publicado: Springer Netherlands 2016
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC5065598/
https://ncbi.nlm.nih.gov/pubmed/27553181
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-016-9973-6
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