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Browsing Research Articles (Paediatrics and Child Health) by Author "AlSayed, Moeenaldeen D."

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Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio a syndrome in an open-label, multi-center, phase 3 extension study

Hendriksz, Christian J.; Parini, Rossella; AlSayed, Moeenaldeen D.; Raiman, Julian; Giugliani, Roberto; Mitchell, John J.; Burton, Barbara K.; Guelbert, Norberto; Stewart, Fiona J.; Hughes, Derralynn A.; Matousek, Robert; Hawley, Sara M.; Decker, Celeste; Harmatz, Paul R. (Elsevier, 2018-02)

BACKGROUND : Long-term safety and efficacy of elosulfase alfa enzyme replacement therapy (ERT) were assessed in 173 patients with Morquio A syndrome (mucopolysaccharidosis IVA) in a 96-week, open-label, multi-center, phase ...

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