{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,28]],"date-time":"2026-02-28T09:50:38Z","timestamp":1772272238452,"version":"3.50.1"},"reference-count":25,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2013,10,10]],"date-time":"2013-10-10T00:00:00Z","timestamp":1381363200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2014,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To evaluate the efficacy and safety of two dose levels of galsulfase (Naglazyme\u00ae) in infants with MPS VI.<\/jats:p><\/jats:sec><jats:sec><jats:title>Study design<\/jats:title><jats:p>This was a phase 4, multicenter, multinational, open\u2010label, two\u2010dose level study. Subjects were randomized 1:1 to receive weekly infusions of 1.0 or 2.0 mg\/kg of galsulfase for a minimum of 52 weeks. Progression of skeletal dysplasia was determined by monitoring physical appearance, radiographic changes, and growth. Urinary glycosaminoglycan (GAG) levels, gross and fine motor function, cardiac function, vision, hearing, and health resource utilization were evaluated. Safety assessments were performed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Four infants (aged 3.3\u201312.7 months) participated in the study. Galsulfase was well tolerated at 1.0 and 2.0 mg\/kg\/week dose levels with no drug\u2010related serious adverse events. Two subjects experienced a total of four possible treatment\u2010related adverse events which were all considered mild. Length and weight remained within age\u2010expected norms. Skeletal abnormalities continued to progress in all subjects. High baseline urinary GAG levels (mean: 870 \u03bcg\/mg creatinine) decreased by approximately 70 %; these reduced levels were maintained (mean: 220 \u03bcg\/mg creatinine at week 52) despite the development of anti\u2010galsulfase antibodies. Hearing, cardiac function, hepatosplenomegaly, and facial dysmorphism stabilized or improved, but corneal clouding progressed. There was no clear difference in safety or efficacy between the two doses.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Galsulfase at two dose levels was safe and well tolerated in infants. Normal growth was maintained but skeletal abnormalities continued to progress. Urinary GAG levels decreased with treatment. Early initiation of galsulfase may prevent or slow progression of some disease manifestations.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s10545-013-9654-7","type":"journal-article","created":{"date-parts":[[2013,10,9]],"date-time":"2013-10-09T13:00:14Z","timestamp":1381323614000},"page":"277-287","source":"Crossref","is-referenced-by-count":35,"title":["Galsulfase (Naglazyme\u00ae) therapy in infants with mucopolysaccharidosis VI"],"prefix":"10.1002","volume":"37","author":[{"given":"Paul R.","family":"Harmatz","sequence":"first","affiliation":[{"name":"Children's Hospital &amp; Research Center Oakland 747 52nd Street Oakland CA USA"}]},{"given":"Paula","family":"Garcia","sequence":"additional","affiliation":[{"name":"Hospital Pedi\u00e1trico de Coimbra Coimbra Portugal"}]},{"given":"Nathalie","family":"Guffon","sequence":"additional","affiliation":[{"name":"H\u00f4pital Femme M\u00e8re Enfant Lyon France"}]},{"given":"Linda M.","family":"Randolph","sequence":"additional","affiliation":[{"name":"Children's Hospital Los Angeles Los Angeles CA USA"}]},{"given":"Ren\u00e9e","family":"Shediac","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc. Novato CA USA"}]},{"given":"Elizabeth","family":"Braunlin","sequence":"additional","affiliation":[{"name":"University of Minnesota Minneapolis MN USA"}]},{"given":"Ralph S.","family":"Lachman","sequence":"additional","affiliation":[{"name":"International Skeletal Dysplasia Registry Cedars\u2010Sinai Medical Center Los Angeles CA USA"}]},{"given":"Celeste","family":"Decker","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc. 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