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dc.contributor.authorOwens, Penny-
dc.contributor.otherBhattacharya, K.-
dc.contributor.otherBalasubramaniam, S.-
dc.contributor.otherMurray, K.-
dc.contributor.otherPeters, H.-
dc.contributor.otherKetteridge, D.-
dc.contributor.otherInwood, A.-
dc.contributor.otherLee, J.-
dc.contributor.otherEllaway, C.-
dc.contributor.otherWong, M.-
dc.description.abstractAbstract The safety and efficacy of elosulfase alfa were evaluated in a multicenter, open-label, phase 3b study in Australian Morquio A patients, consisting of a 49-week initial phase and an extension phase until elosulfase alfa was government funded. Thirteen patients (1-27 years) were enrolled. No new safety concerns were identified over 138 weeks. Most drug-related adverse events were mild or moderate in severity; none led to study discontinuation. After 49 weeks of treatment, median improvements from baseline were seen in the 6-minute walk test (+41.0 m), 3-minute stair climb test (+14.0 stairs/min), forced vital capacity (+16.4%), forced expiratory volume in 1 second (+14.1%), urine keratan sulfate (-7.1 µg/mg creatinine), and pain intensity. Growth, cardiac function, sleep, and quality of life results were mixed or stable. These results provide further evidence of the acceptable safety/tolerability profile of elosulfase alfa. The improvements in endurance, pulmonary function, and pain support findings from previous studies.en
dc.titleSafety and Efficacy of Elosulfase Alfa in Australian Patients with Morquio a Syndrome: A Phase 3b Studyen
dc.typeJournal Articleen
dc.description.affiliatesCentral Coast Local Health Districten
dc.identifier.journaltitleJournal of Inborn Errors of Metabolism and Screeningen
dc.type.studyortrialMulticentre Studiesen
item.openairetypeJournal Article-
item.fulltextNo Fulltext-
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