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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Pubblicato in:J Inherit Metab Dis
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Springer Netherlands 2016
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Original Article
Accesso online: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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