{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,20]],"date-time":"2026-09-20T18:23:05Z","timestamp":1789928585790,"version":"4.0.1"},"reference-count":0,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2010,1,1]],"date-time":"2010-01-01T00:00:00Z","timestamp":1262304000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Journal of Pediatric Rehabilitation Medicine"],"published-print":{"date-parts":[[2010,5]]},"abstract":"<jats:p>Background and Methods: Growth failure is characteristic of\n\t\t\t untreated mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome). \n\t\t\t Growth was studied in fifty-six MPS VI patients (5 to 29 years old) prior to\n\t\t\t and for up to 240 weeks of weekly infusions of recombinant human arylsulfatase\n\t\t\t B (rhASB) at 1 mg\/kg during Phase 1\/2, Phase 2, Phase 3 or Phase 3 Extension\n\t\t\t clinical trials. Height, weight, and Tanner stage data were collected. Pooled\n\t\t\t data were analyzed to determine mean height increase by treatment week, growth\n\t\t\t impacts of pubertal status, baseline urinary GAG, and age at treatment\n\t\t\t initiation. Growth rate for approximately 2 years prior to and following\n\t\t\t treatment initiation was analyzed using longitudinal modeling.<\/jats:p>\n                  <jats:p>Results: Mean height increased by 2.9 cm after 48 weeks and 4.3 cm after 96 weeks on\n\t\t\t enzyme replacement therapy (ERT). Growth on ERT was not correlated with\n\t\t\t baseline urinary GAG. Patients under 16~years of age showed greatest increases\n\t\t\t in height on treatment. Model results based on pooled data showed significant\n\t\t\t improvement in growth rate during 96~weeks of ERT when compared to the\n\t\t\t equivalent pretreatment time period. Delayed pubertal onset or progression was\n\t\t\t noted in 10 patients entering the clinical trials; all of whom showed\n\t\t\t progression of at least one Tanner stage during 2 years on ERT, and 6 of whom\n\t\t\t (60%) completed puberty.<\/jats:p>\n                  <jats:p>Conclusion: Analysis of mean height by\n\t\t\t treatment week and longitudinal modeling demonstrate significant increase in\n\t\t\t height and growth rate in MPS VI patients receiving long-term ERT. This impact\n\t\t\t was greatest in patients aged below 16 years. Height increase may result from\n\t\t\t bone growth and\/or reduction in joint contractures. Bone growth and resolution\n\t\t\t of delayed puberty may be related to improvements in general health, bone cell\n\t\t\t health, nutrition, endocrine gland function and reduced inflammation.<\/jats:p>","DOI":"10.3233\/prm-2010-0113","type":"journal-article","created":{"date-parts":[[2024,12,4]],"date-time":"2024-12-04T12:23:23Z","timestamp":1733315003000},"page":"89-100","source":"Crossref","is-referenced-by-count":4,"title":["Enzyme replacement therapy for mucopolysaccharidosis VI: Growth\t\t\t and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase"],"prefix":"10.1177","volume":"3","author":[{"given":"Celeste","family":"Decker","sequence":"first","affiliation":[{"name":"BioMarin Pharmaceutical Inc, Novato, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zi-Fan","family":"Yu","sequence":"additional","affiliation":[{"name":"Statistics Collaborative, Inc., Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberto","family":"Giugliani","sequence":"additional","affiliation":[{"name":"Servi\u00e7o de Gen\u00e9tica M\u00e9dica\/ HCPA and\r\t\t\t Department of Genetics\/UFRGS, Porto Alegre, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ida Vanessa D.","family":"Schwartz","sequence":"additional","affiliation":[{"name":"Servi\u00e7o de Gen\u00e9tica M\u00e9dica\/ HCPA and\r\t\t\t Department of Genetics\/UFRGS, Porto Alegre, Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathalie","family":"Guffon","sequence":"additional","affiliation":[{"name":"H\u00f4pital Femme M\u00e8re Enfant, Service Maladies\r\t\t\t M\u00e9taboliques, BRON, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elisa Le\u00e3o","family":"Teles","sequence":"additional","affiliation":[{"name":"Unidade de Doen\u00e7as Metab\u00f3licas, Departamento\r\t\t\t Pediatria, Hospital de Sao Jo\u00e3o, Porto, Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. Clara S\u00e1","family":"Miranda","sequence":"additional","affiliation":[{"name":"Unidade de Biologia do Lisossoma e Peroxisoma,\r\t\t\t Instituto de Biologia Molecular e Celular, Porto, Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J. Edmond","family":"Wraith","sequence":"additional","affiliation":[{"name":"Royal Manchester Children's Hospital, Manchester,\r\t\t\t UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Beck","sequence":"additional","affiliation":[{"name":"Children's Hospital, University of Mainz,\r\t\t\t Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laila","family":"Arash","sequence":"additional","affiliation":[{"name":"Children's Hospital, University of Mainz,\r\t\t\t Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maurizio","family":"Scarpa","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Padova,\r\t\t\t Padova, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David","family":"Ketteridge","sequence":"additional","affiliation":[{"name":"SA Pathology at Women's and Children's Hospital, North\r\t\t\t Adelaide, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John J.","family":"Hopwood","sequence":"additional","affiliation":[{"name":"Department of Genetic Medicine, Women's and Children's\r\t\t\t Hospital Adelaide, North Adelaide, Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Plecko","sequence":"additional","affiliation":[{"name":"University Klinik f\u00fcr Kinder und Jugendheilkunde, Graz,\r\t\t\t Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Steiner","sequence":"additional","affiliation":[{"name":"epartments of Pediatrics and Molecular and Medical\r\t\t\t Genetics, Oregon Health &amp;} Science University, Portland, OR, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chester B.","family":"Whitley","sequence":"additional","affiliation":[{"name":"University of Minnesota Medical School, Minneapolis,\r\t\t\t MN, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paige","family":"Kaplan","sequence":"additional","affiliation":[{"name":"Children's Hospital of Philadelphia, Philadelphia, PA,\r\t\t\t USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stuart J.","family":"Swiedler","sequence":"additional","affiliation":[{"name":"BioMarin Pharmaceutical Inc, Novato, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan","family":"Conrad","sequence":"additional","affiliation":[{"name":"Children's Hospital &amp; Research Center Oakland,\r\t\t\t Oakland, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"family":"Paul Harmatz for the MPS VI Study Group","sequence":"additional","affiliation":[{"name":"Children's Hospital &amp; Research Center Oakland,\r\t\t\t Oakland, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2010,1]]},"container-title":["Journal of Pediatric Rehabilitation Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.3233\/PRM-2010-0113","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.3233\/PRM-2010-0113","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T04:38:19Z","timestamp":1777610299000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.3233\/PRM-2010-0113"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2010,1]]},"references-count":0,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2010,5]]}},"alternative-id":["10.3233\/PRM-2010-0113"],"URL":"https:\/\/doi.org\/10.3233\/prm-2010-0113","relation":{"is-cited-by":[{"id-type":"doi","id":"10.1186\/s13256-021-03240-3","asserted-by":"object"}]},"ISSN":["1874-5393","1875-8894"],"issn-type":[{"value":"1874-5393","type":"print"},{"value":"1875-8894","type":"electronic"}],"subject":[],"published":{"date-parts":[[2010,1]]}}}