{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T15:28:58Z","timestamp":1791300538506,"version":"4.1.0"},"reference-count":30,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2008,4,1]],"date-time":"2008-04-01T00:00:00Z","timestamp":1207008000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2013,7,21]],"date-time":"2013-07-21T00:00:00Z","timestamp":1374364800000},"content-version":"vor","delay-in-days":1937,"URL":"https:\/\/www.elsevier.com\/open-access\/userlicense\/1.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Current Therapeutic Research"],"published-print":{"date-parts":[[2008,4]]},"DOI":"10.1016\/j.curtheres.2008.04.002","type":"journal-article","created":{"date-parts":[[2008,5,29]],"date-time":"2008-05-29T08:47:13Z","timestamp":1212050833000},"page":"166-179","source":"Crossref","is-referenced-by-count":93,"title":["Genistin-rich soy isoflavone extract in substrate reduction therapy for Sanfilippo syndrome: An open-label, pilot study in 10 pediatric patients"],"prefix":"10.1016","volume":"69","author":[{"given":"Ewa","family":"Piotrowska","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanna","family":"Jak\u00f3bkiewicz-Banecka","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Tylki-Szymanska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Liberek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Agnieszka","family":"Maryniak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcelina","family":"Malinowska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Czartoryska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ewa","family":"Puk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna","family":"Kloska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tomasz","family":"Liberek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylwia","family":"Baranska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alicja","family":"Wegrzyn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Grzegorz","family":"Wegrzyn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.curtheres.2008.04.002_bib1","series-title":"The Metabolic and Molecular Bases of Inherited Disease","first-page":"3421","article-title":"The mucopolysaccharidoses","author":"Neufeld","year":"2001"},{"key":"10.1016\/j.curtheres.2008.04.002_bib2","doi-asserted-by":"crossref","first-page":"986","DOI":"10.1016\/j.mehy.2003.12.027","article-title":"A general model for genetic regulation of turn-over of glycosaminoglycans suggests a possible procedure for prediction of severity and clinical progress of mucopolysaccharidoses","volume":"62","author":"Wegrzyn","year":"2004","journal-title":"Med Hypotheses"},{"key":"10.1016\/j.curtheres.2008.04.002_bib3","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1056\/NEJM200101183440304","article-title":"Enzyme-replacement therapy in mucopolysaccharidosis I","volume":"344","author":"Kakkis","year":"2001","journal-title":"N Engl J Med"},{"key":"10.1016\/j.curtheres.2008.04.002_bib4","doi-asserted-by":"crossref","first-page":"581","DOI":"10.1016\/j.jpeds.2004.01.046","article-title":"Enzyme replacement therapy for mucopolysaccharidosis I: A randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase)","volume":"144","author":"Wraith","year":"2004","journal-title":"J Pediatr"},{"key":"10.1016\/j.curtheres.2008.04.002_bib5","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/j.ymgme.2006.08.007","article-title":"A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years","volume":"90","author":"Sifuentes","year":"2007","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.curtheres.2008.04.002_bib6","first-page":"465","article-title":"A phase II\/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome) [published correction appears in Genet Med","volume":"8","author":"Muenzer","year":"2006","journal-title":"2006;8:599]. Genet Med"},{"key":"10.1016\/j.curtheres.2008.04.002_bib7","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1016\/j.jpeds.2005.12.014","volume":"148","author":"Harmatz","year":"2006","journal-title":"J Pediatr"},{"key":"10.1016\/j.curtheres.2008.04.002_bib8","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1016\/j.ymgme.2004.07.003","article-title":"Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I","volume":"83","author":"Kakkis","year":"2004","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.curtheres.2008.04.002_bib9","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1016\/j.ymgme.2006.12.012","article-title":"Intrathecal enzyme replacement therapy: Successful treatment of brain disease via the cerebrospinal fluid","volume":"91","author":"Dickson","year":"2007","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.curtheres.2008.04.002_bib10","doi-asserted-by":"crossref","first-page":"733","DOI":"10.2165\/00003495-200262050-00002","article-title":"New prospects for the treatment of lysosomal storage diseases","volume":"62","author":"Schiffmann","year":"2002","journal-title":"Drugs"},{"key":"10.1016\/j.curtheres.2008.04.002_bib11","doi-asserted-by":"crossref","first-page":"846","DOI":"10.1038\/sj.ejhg.5201623","article-title":"Genistein-mediated inhibition of glycosaminoglycan synthesis as a basis for gene expression-targeted isoflavone therapy for mucopolysaccharidoses","volume":"14","author":"Piotrowska","year":"2006","journal-title":"Eur J Hum Genet"},{"key":"10.1016\/j.curtheres.2008.04.002_bib12","doi-asserted-by":"crossref","first-page":"4787","DOI":"10.1074\/jbc.272.8.4787","article-title":"Hyaluronan synthesis by mouse cumulus cells is regulated by interactions between follicle-stimulating hormone (or epidermal growth factor) and a soluble oocyte factor (or transforming growth factor beta1)","volume":"272","author":"Tirone","year":"1997","journal-title":"J Biol Chem"},{"key":"10.1016\/j.curtheres.2008.04.002_bib13","doi-asserted-by":"crossref","first-page":"419","DOI":"10.1016\/0012-1606(87)90306-X","article-title":"Epidermal growth factor potentiates the induction of ornithine decarboxylase activity by prostaglandins in embryonic palate mesenchymal cells: Effects on cell proliferation and glycosaminoglycan synthesis","volume":"122","author":"Pisano","year":"1987","journal-title":"Dev Biol"},{"key":"10.1016\/j.curtheres.2008.04.002_bib14","doi-asserted-by":"crossref","first-page":"5592","DOI":"10.1016\/S0021-9258(18)45614-1","article-title":"Genistein, a specific inhibitor of tyrosine-specific protein kinases","volume":"262","author":"Akiyama","year":"1987","journal-title":"J Biol Chem"},{"issue":"Suppl 1","key":"10.1016\/j.curtheres.2008.04.002_bib15","doi-asserted-by":"crossref","first-page":"1418S","DOI":"10.1093\/ajcn\/68.6.1418S","article-title":"Mechanisms of action of the soy isoflavone genistein: Emerging role for its effects via transforming growth factor beta signaling pathways","volume":"68","author":"Kim","year":"1998","journal-title":"Am J Clin Nutr"},{"key":"10.1016\/j.curtheres.2008.04.002_bib16","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1016\/j.chroma.2004.10.048","article-title":"Concurrent measurement of unbound genistein in the blood, brain and bile of anesthetized rats using microdialysis and its pharmacokinetic application","volume":"1073","author":"Tsai","year":"2005","journal-title":"J Chromatogr A"},{"key":"10.1016\/j.curtheres.2008.04.002_bib17","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1203\/01.pdr.0000233037.00707.da","article-title":"Inhibition of glycosaminoglycan synthesis using rhodamine B in a mouse model of mucopolysaccharidosis type IIIA","volume":"60","author":"Roberts","year":"2006","journal-title":"Pediatr Res"},{"key":"10.1016\/j.curtheres.2008.04.002_bib18","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1093\/ajcn\/76.5.1126","article-title":"Safety and pharmacokinetics of purified soy isoflavones: Single-dose administration to postmenopausal women","volume":"76","author":"Bloedon","year":"2002","journal-title":"Am J Clin Nutr"},{"key":"10.1016\/j.curtheres.2008.04.002_bib19","doi-asserted-by":"crossref","first-page":"1461","DOI":"10.1016\/j.fct.2005.02.017","article-title":"Subchronic and chronic safety studies with genistein in dogs","volume":"43","author":"McClain","year":"2005","journal-title":"Food Chem Toxicol"},{"key":"10.1016\/j.curtheres.2008.04.002_bib20","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1007\/BF02850186","article-title":"Safety, tolerability, and pharmacokinetics of single ascending doses of synthetic genistein (Bonistein) in healthy volunteers","volume":"22","author":"Ullmann","year":"2005","journal-title":"Adv Ther"},{"key":"10.1016\/j.curtheres.2008.04.002_bib21","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1007\/s00431-007-0462-7","article-title":"Abnormalities in the hair morphology of patients with some but not all types of mucopolysaccharidoses","volume":"167","author":"Malinowska","year":"2008","journal-title":"Eur J Pediatr"},{"key":"10.1016\/j.curtheres.2008.04.002_bib22","doi-asserted-by":"crossref","first-page":"173","DOI":"10.1177\/088307389601100301","article-title":"Use of a brief assessment examination in a study of subacute sclerosing panencephalitis","volume":"11","author":"Nester","year":"1996","journal-title":"J Child Neurol"},{"key":"10.1016\/j.curtheres.2008.04.002_bib23","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1186\/1471-2431-5-47","article-title":"Subacute sclerosing panencephalitis: Results of the Canadian Paediatric Surveillance Program and review of the literature","volume":"5","author":"Campbell","year":"2005","journal-title":"BMC Pediatr"},{"key":"10.1016\/j.curtheres.2008.04.002_bib24","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1002\/ajmg.a.31021","article-title":"Changes in hair morphology of mucopolysaccharidosis I patients treated with recombinant human alpha-L-iduronidase (laronidase, Aldurazyme)","volume":"139","author":"Kloska","year":"2005","journal-title":"Am J Med Genet A"},{"key":"10.1016\/j.curtheres.2008.04.002_bib25","doi-asserted-by":"crossref","first-page":"743","DOI":"10.1007\/s10545-005-0069-y","article-title":"Heparan sulfate levels in mucopolysaccharidoses and mucolipidoses","volume":"28","author":"Tomatsu","year":"2005","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.curtheres.2008.04.002_bib26","first-page":"1925","article-title":"et al","volume":"143","author":"Wegrzyn","year":"2007","journal-title":"Rapid deterioration of a patient with mucopolysaccharidosis type I during interruption of enzyme replacement therapy. Am J Med Genet"},{"key":"10.1016\/j.curtheres.2008.04.002_bib27","doi-asserted-by":"crossref","first-page":"80","DOI":"10.2307\/3001968","article-title":"Individual comparisons by ranking methods","volume":"1","author":"Wilcoxon","year":"1945","journal-title":"Biometr Bull"},{"key":"10.1016\/j.curtheres.2008.04.002_bib28","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1016\/S1474-4422(07)70194-1","article-title":"Miglustat for treatment of Niemann-Pick C disease: A randomised controlled study","volume":"6","author":"Patterson","year":"2007","journal-title":"Lancet Neurol"},{"key":"10.1016\/j.curtheres.2008.04.002_bib29","doi-asserted-by":"crossref","first-page":"748","DOI":"10.1016\/S1474-4422(07)70198-9","article-title":"A first therapy for Niemann-Pick C","volume":"6","author":"Erickson","year":"2007","journal-title":"Lancet Neurol"},{"key":"10.1016\/j.curtheres.2008.04.002_bib30","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1007\/BF03195237","article-title":"Substrate deprivation therapy: A new hope for patients suffering from neuronopathic forms of inherited lysosomal storage diseases","volume":"48","author":"Jak\u00f3bkiewicz-Banecka","year":"2007","journal-title":"J Appl Genet"}],"container-title":["Current Therapeutic Research"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0011393X08000362?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0011393X08000362?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2021,9,11]],"date-time":"2021-09-11T07:31:48Z","timestamp":1631345508000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0011393X08000362"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2008,4]]},"references-count":30,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2008,4]]}},"alternative-id":["S0011393X08000362"],"URL":"https:\/\/doi.org\/10.1016\/j.curtheres.2008.04.002","relation":{},"ISSN":["0011-393X"],"issn-type":[{"value":"0011-393X","type":"print"}],"subject":[],"published":{"date-parts":[[2008,4]]}}}