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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Bibliographic Details
Published in: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.
Format: Artigo
Language:Inglês
Published: Springer Netherlands 2016
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Original Article
Online Access: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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