{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,21]],"date-time":"2026-04-21T05:48:24Z","timestamp":1776750504000,"version":"3.51.2"},"reference-count":32,"publisher":"Wiley","issue":"12","license":[{"start":{"date-parts":[[2019,10,22]],"date-time":"2019-10-22T00:00:00Z","timestamp":1571702400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","award":["MPS I International Registry"],"award-info":[{"award-number":["MPS I International Registry"]}],"id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100013995","name":"Sanofi Genzyme","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013995","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J of Med Genetics Pt A"],"published-print":{"date-parts":[[2019,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Mucopolysaccharidosis Type I (MPS I), caused by deficiency of \u03b1\u2010L\u2010iduronidase results in progressive, multisystemic disease with a broad phenotypic spectrum including patients with severe (Hurler syndrome) to attenuated (Hurler\u2013Scheie and Scheie syndromes) disease. Disordered growth is common with either phenotype. The study objectives were to construct sex\u2010 and age\u2010specific estimated length\/height and head circumference growth curves for untreated individuals with severe and attenuated disease and compare them with clinical reference standards. Untreated individuals in the MPS I Registry with at least one observation for length\/height and\/or head circumference and assigned phenotype as of May 2017 were included. Median growth for 463 untreated individuals with severe disease deviated from reference growth curves by ~6\u2009months of age and fell below the third percentile by 4\u2009years of age. Median head circumference was above reference curves from 3 to 4 months through 3\u2009years of age. Among 207 individuals with untreated attenuated disease, median height fell below the third percentile by 9\u2009years of age with divergence from reference curves by 2\u2009years of age. MPS I\u2010specific growth curves will be useful in evaluation of long\u2010term outcomes of therapeutics interventions and will provide a foundation for understanding the pathogenesis of skeletal disease in MPS I.<\/jats:p>","DOI":"10.1002\/ajmg.a.61378","type":"journal-article","created":{"date-parts":[[2019,10,22]],"date-time":"2019-10-22T16:42:27Z","timestamp":1571762547000},"page":"2425-2432","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["Growth patterns for untreated individuals with MPS I: Report from the international MPS I registry"],"prefix":"10.1002","volume":"179","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5364-3366","authenticated-orcid":false,"given":"David","family":"Viskochil","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Division of Medical Genetics University of Utah School of Medicine  Salt Lake City Utah"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lorne A.","family":"Clarke","sequence":"additional","affiliation":[{"name":"British Columbia Children's Hospital Research Institute University of British Columbia  Vancouver British Columbia Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Luisa","family":"Bay","sequence":"additional","affiliation":[{"name":"Hospital Nacional de Pediatr\u00eda J. 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