{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T14:04:54Z","timestamp":1792159494470,"version":"4.4.0"},"reference-count":33,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2014,5,9]],"date-time":"2014-05-09T00:00:00Z","timestamp":1399593600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2014,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To assess the efficacy and safety of enzyme replacement therapy (ERT) with BMN 110 (elosulfase alfa) in patients with Morquio A syndrome (mucopolysaccharidosis IVA).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Patients with Morquio A aged \u22655 years (<jats:italic>N<\/jats:italic>\u2009=\u2009176) were randomised (1:1:1) to receive elosulfase alfa 2.0 mg\/kg\/every other week (qow), elosulfase alfa 2.0 mg\/kg\/week (weekly) or placebo for 24 weeks in this phase 3, double\u2010blind, randomised study. The primary efficacy measure was 6\u2010min walk test (6MWT) distance. Secondary efficacy measures were 3\u2010min stair climb test (3MSCT) followed by change in urine keratan sulfate (KS). Various exploratory measures included respiratory function tests. Patient safety was also evaluated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>At week 24, the estimated mean effect on the 6MWT versus placebo was 22.5 m (95 % CI 4.0, 40.9; <jats:italic>P<\/jats:italic>\u2009=\u20090.017) for weekly and 0.5 m (95 % CI \u221217.8, 18.9; <jats:italic>P<\/jats:italic>\u2009=\u20090.954) for qow. The estimated mean effect on 3MSCT was 1.1 stairs\/min (95 % CI \u22122.1, 4.4; <jats:italic>P<\/jats:italic>\u2009=\u20090.494) for weekly and \u22120.5 stairs\/min (95 % CI \u22123.7, 2.8; <jats:italic>P<\/jats:italic>\u2009=\u20090.778) for qow. Normalised urine KS was reduced at 24 weeks in both regimens. In the weekly dose group, 22.4 % of patients had adverse events leading to an infusion interruption\/discontinuation requiring medical intervention (only 1.3 % of all infusions in this group) over 6 months. No adverse events led to permanent treatment discontinuation.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Elosulfase alfa improved endurance as measured by the 6MWT in the weekly but not qow dose group, did not improve endurance on the 3MSCT, reduced urine KS, and had an acceptable safety profile.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s10545-014-9715-6","type":"journal-article","created":{"date-parts":[[2014,5,8]],"date-time":"2014-05-08T14:17:36Z","timestamp":1399558656000},"page":"979-990","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":191,"title":["Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo\u2010controlled study"],"prefix":"10.1002","volume":"37","author":[{"given":"Christian J.","family":"Hendriksz","sequence":"first","affiliation":[{"name":"Birmingham Children's Hospital NHS Foundation Trust Birmingham UK"},{"name":"Manchester Academic Health Science Centre, The Mark Holland Metabolic Unit Salford Royal Foundation NHS Trust Ladywell NW2\u2010 2nd Floor Room 107, Salford Manchester M6 8HD UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Burton","sequence":"additional","affiliation":[{"name":"Ann and Robert H. 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