Ontology highlight
ABSTRACT:
SUBMITTER: Hendriksz CJ
PROVIDER: S-EPMC5065598 | biostudies-literature | 2016 Nov
REPOSITORIES: biostudies-literature
Hendriksz Christian J CJ Berger Kenneth I KI Parini Rossella R AlSayed Moeenaldeen D MD Raiman Julian J Giugliani Roberto R Mitchell John J JJ Burton Barbara K BK Guelbert Norberto N Stewart Fiona F Hughes Derralynn A DA Matousek Robert R Jurecki Elaina E Decker Celeste C Harmatz Paul R PR
Journal of inherited metabolic disease 20160823 6
<h4>Objective</h4>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.<h4>Methods</h4>In part 1 of MOR-005, patients initially randomized to ERT in the 24-week pivotal study (MOR-004) remained on their regimen (2.0 mg/kg/week or every other week); placebo patients were re-randomized to one of the two regimens. During part 2, all patients recei ...[more]