{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T10:23:03Z","timestamp":1791973383821,"version":"4.3.4"},"reference-count":45,"publisher":"Elsevier BV","issue":"8","license":[{"start":{"date-parts":[[2005,8,1]],"date-time":"2005-08-01T00:00:00Z","timestamp":1122854400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Clinical Therapeutics"],"published-print":{"date-parts":[[2005,8]]},"DOI":"10.1016\/j.clinthera.2005.08.004","type":"journal-article","created":{"date-parts":[[2005,10,2]],"date-time":"2005-10-02T11:07:49Z","timestamp":1128251269000},"page":"1215-1227","source":"Crossref","is-referenced-by-count":119,"title":["An open-label, noncomparative study of miglustat in type I Gaucher disease: Efficacy and tolerability over 24 months of treatment"],"prefix":"10.1016","volume":"27","author":[{"given":"Gregory M.","family":"Pastores","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie L.","family":"Barnett","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edwin H.","family":"Kolodny","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.clinthera.2005.08.004_bib1","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1016\/S0950-3536(97)80033-9","article-title":"Gaucher's disease: Clinical features and natural history","volume":"10","author":"Cox","year":"1997","journal-title":"Baillieres Clin Haematol"},{"key":"10.1016\/j.clinthera.2005.08.004_bib2","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1097\/00005792-199211000-00002","article-title":"Gaucher disease. Clinical, laboratory, radiologic, and genetic features of 53 patients","volume":"71","author":"Zimran","year":"1992","journal-title":"Medicine (Baltimore)"},{"key":"10.1016\/j.clinthera.2005.08.004_bib3","series-title":"Cerebral Sphingolipidoses","first-page":"395","article-title":"Genetics of the sphingolipidoses","author":"Knudson","year":"1962"},{"key":"10.1016\/j.clinthera.2005.08.004_bib4","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1093\/qjmed\/hch036","article-title":"Twin pairs showing discordance of phenotype in adult Gaucher's disease","volume":"97","author":"Lachmann","year":"2004","journal-title":"QJM"},{"key":"10.1016\/j.clinthera.2005.08.004_bib5","doi-asserted-by":"crossref","first-page":"1464","DOI":"10.1056\/NEJM199105233242104","article-title":"Replacement therapy for inherited enzyme deficiency\u2014macrophagetargeted glucocerebrosidase for Gaucher's disease","volume":"324","author":"Barton","year":"1991","journal-title":"N Engl J Med"},{"key":"10.1016\/j.clinthera.2005.08.004_bib6","doi-asserted-by":"crossref","first-page":"33","DOI":"10.7326\/0003-4819-122-1-199501010-00005","article-title":"Enzyme therapy in type 1 Gaucher disease: Comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources","volume":"122","author":"Grabowski","year":"1995","journal-title":"Ann Intern Med"},{"key":"10.1016\/j.clinthera.2005.08.004_bib7","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1016\/S0002-9343(02)01150-6","article-title":"Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: A report from the Gaucher Registry","volume":"113","author":"Weinreb","year":"2002","journal-title":"Am J Med"},{"issue":"Suppl 5","key":"10.1016\/j.clinthera.2005.08.004_bib8","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1053\/j.seminhematol.2004.07.009","article-title":"Therapeutic goals in the treatment of Gaucher disease","volume":"41","author":"Pastores","year":"2004","journal-title":"Semin Hematol"},{"key":"10.1016\/j.clinthera.2005.08.004_bib9","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1182\/blood-2003-11-3854","article-title":"Gaucher disease: Alendronate disodium improves bone mineral density in adults receiving enzyme therapy","volume":"104","author":"Wenstrup","year":"2004","journal-title":"Blood"},{"key":"10.1016\/j.clinthera.2005.08.004_bib10","doi-asserted-by":"crossref","first-page":"408","DOI":"10.1182\/blood.V82.2.408.408","article-title":"Enzyme therapy in Gaucher disease type 1: Dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months","volume":"82","author":"Pastores","year":"1993","journal-title":"Blood"},{"key":"10.1016\/j.clinthera.2005.08.004_bib11","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1006\/bcmd.2001.0477","article-title":"Early-onset severe neurological involvement and D409H homozygosity in Gaucher disease: Outcome of enzyme replacement therapy","volume":"28","author":"Michelakakis","year":"2002","journal-title":"Blood Cells Mol Dis"},{"key":"10.1016\/j.clinthera.2005.08.004_bib12","doi-asserted-by":"crossref","first-page":"629","DOI":"10.1023\/B:BOLI.0000005619.14180.5c","article-title":"A model of neuronopathic Gaucher disease","volume":"26","author":"Campbell","year":"2003","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.clinthera.2005.08.004_bib13","doi-asserted-by":"crossref","first-page":"513","DOI":"10.1023\/A:1025902113005","article-title":"The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: A position statement","volume":"26","author":"Cox","year":"2003","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.clinthera.2005.08.004_bib14","doi-asserted-by":"crossref","first-page":"1402","DOI":"10.1182\/blood.V82.5.1402.1402","article-title":"Antibody response in patients with Gaucher disease after repeated infusion with macrophage-targeted glucocerebrosidase","volume":"82","author":"Richards","year":"1993","journal-title":"Blood"},{"key":"10.1016\/j.clinthera.2005.08.004_bib15","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1006\/bcmd.1999.0231","article-title":"Anaphylactoid reaction to imiglucerase, but not to alglucerase, in a type I Gaucher patient","volume":"25","author":"Aviner","year":"1999","journal-title":"Blood Cells Mol Dis"},{"key":"10.1016\/j.clinthera.2005.08.004_bib16","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/S1079-9796(03)00012-3","article-title":"Enzyme therapy of Gaucher disease: Clinical and biochemical changes during production of and tolerization for neutralizing antibodies","volume":"30","author":"Zhao","year":"2003","journal-title":"Blood Cells Mol Dis"},{"key":"10.1016\/j.clinthera.2005.08.004_bib17","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1016\/0009-3084(80)90057-2","article-title":"Analogs of ceramide that inhibit glucocerebroside synthetase in mouse brain","volume":"26","author":"Vunnam","year":"1980","journal-title":"Chem Phys Lipids"},{"key":"10.1016\/j.clinthera.2005.08.004_bib18","doi-asserted-by":"crossref","first-page":"8362","DOI":"10.1016\/S0021-9258(17)37202-2","article-title":"N-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis","volume":"269","author":"Platt","year":"1994","journal-title":"J Biol Chem"},{"key":"10.1016\/j.clinthera.2005.08.004_bib19","doi-asserted-by":"crossref","first-page":"1481","DOI":"10.1016\/S0140-6736(00)02161-9","article-title":"Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis","volume":"355","author":"Cox","year":"2000","journal-title":"Lancet"},{"key":"10.1016\/j.clinthera.2005.08.004_bib20","doi-asserted-by":"crossref","first-page":"641","DOI":"10.1023\/B:BOLI.0000005623.60471.51","article-title":"A neurological symptom survey of patients with type I Gaucher disease","volume":"26","author":"Pastores","year":"2003","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.clinthera.2005.08.004_bib21","series-title":"Zavesca (miglustat) capsules, 100 mg [package insert]","year":"2003"},{"key":"10.1016\/j.clinthera.2005.08.004_bib22","first-page":"373","article-title":"Ethical principles for medical research involving human subjects","volume":"79","year":"2001","journal-title":"Bull World Health Organ"},{"issue":"Suppl 1","key":"10.1016\/j.clinthera.2005.08.004_bib23","doi-asserted-by":"crossref","first-page":"A13","DOI":"10.1259\/bjr.75.suppl_1.750013","article-title":"Imaging and quantifying skeletal involvement in Gaucher disease","volume":"75","author":"Maas","year":"2002","journal-title":"Brit J Radiol"},{"key":"10.1016\/j.clinthera.2005.08.004_bib24","first-page":"1","article-title":"Assessment of fracture risk and its application to screening for post-menopausal osteoporosis. Report of a WHO Study Group","volume":"843","year":"1994","journal-title":"World Health Organ Tech Rep Ser"},{"key":"10.1016\/j.clinthera.2005.08.004_bib25","doi-asserted-by":"crossref","first-page":"1801","DOI":"10.1002\/jbmr.5650111125","article-title":"Bone density in type 1 Gaucher disease","volume":"11","author":"Pastores","year":"1996","journal-title":"J Bone Miner Res"},{"key":"10.1016\/j.clinthera.2005.08.004_bib26","article-title":"Comparison of fast Fourier transformation and autoregressive modelling as a diagnostic tool in analysis of lower extremity venous signals","author":"Kara","year":"2005","journal-title":"Comput Biol Med."},{"key":"10.1016\/j.clinthera.2005.08.004_bib27","doi-asserted-by":"crossref","first-page":"205","DOI":"10.1016\/S0022-510X(96)00231-6","article-title":"International Cooperative Ataxia Rating Scale for pharmacological assessment of the cerebellar syndrome","volume":"145","author":"Trouillas","year":"1997","journal-title":"J Neurol Sci"},{"key":"10.1016\/j.clinthera.2005.08.004_bib28","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1197\/j.jht.2005.04.012","article-title":"Functional tests to quantify recovery following carpal tunnel release","volume":"18","author":"Rider","year":"2005","journal-title":"J Hand Ther"},{"key":"10.1016\/j.clinthera.2005.08.004_bib29","doi-asserted-by":"crossref","first-page":"189","DOI":"10.1016\/0022-3956(75)90026-6","article-title":"\u201cMini-mental state.\u201d A practical method for grading the cognitive state of patients for the clinician","volume":"12","author":"Folstein","year":"1975","journal-title":"J Psychiatr Res"},{"key":"10.1016\/j.clinthera.2005.08.004_bib30","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1037\/h0061266","article-title":"The Purdue pegboard: Norms and studies of reliability and variability","volume":"32","author":"Tiffin","year":"1948","journal-title":"J Appl Psychol"},{"key":"10.1016\/j.clinthera.2005.08.004_bib31","series-title":"Wechsler Abbreviated Scale of Intelligence Manual","author":"Wechsler","year":"1999"},{"key":"10.1016\/j.clinthera.2005.08.004_bib32","series-title":"Wechsler Adult Intelligence Scale\u2014Third Edition Administration and Scoring Manual","author":"Wechsler","year":"1997"},{"key":"10.1016\/j.clinthera.2005.08.004_bib33","series-title":"Stroop Color and Word Test","author":"Golden","year":"1978"},{"key":"10.1016\/j.clinthera.2005.08.004_bib34","series-title":"The Halstead-Reitan Neuropsychological Test Battery","author":"Reitan","year":"1985"},{"key":"10.1016\/j.clinthera.2005.08.004_bib35","series-title":"Wisconsin Card Sorting Test\u201464 Card Version Professional Manual","author":"Kongs","year":"2000"},{"key":"10.1016\/j.clinthera.2005.08.004_bib36","series-title":"The Assessment of Aphasia and Related Disorders","author":"Goodglass","year":"1983"},{"key":"10.1016\/j.clinthera.2005.08.004_bib37","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1080\/13854049108403297","article-title":"The Hopkins Verbal Learning Test: Development of a new memory test with six equivalent forms","volume":"5","author":"Brandt","year":"1991","journal-title":"Clin Neuropsychol"},{"key":"10.1016\/j.clinthera.2005.08.004_bib38","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1080\/00325481.1972.11713319","article-title":"Screening depressed patients in family practice. A rapid technic","volume":"52","author":"Beck","year":"1972","journal-title":"Postgrad Med"},{"key":"10.1016\/j.clinthera.2005.08.004_bib39","series-title":"Beck Anxiety Inventory","author":"Beck","year":"1990"},{"key":"10.1016\/j.clinthera.2005.08.004_bib40","series-title":"The Boston Naming Test","author":"Kaplan","year":"1983"},{"key":"10.1016\/j.clinthera.2005.08.004_bib41","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1080\/13854048908403285","article-title":"Predicting premorbid IQ: A revision of the National Adult Reading Test","volume":"3","author":"Blair","year":"1989","journal-title":"Clin Neuropsychol"},{"key":"10.1016\/j.clinthera.2005.08.004_bib42","first-page":"206","article-title":"Le test de copie d'une figure complexe","volume":"30","author":"Osterrieth","year":"1944","journal-title":"Arch Psychol"},{"key":"10.1016\/j.clinthera.2005.08.004_bib43","doi-asserted-by":"crossref","first-page":"757","DOI":"10.1023\/B:BOLI.0000045756.54006.17","article-title":"Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease","volume":"27","author":"Elstein","year":"2004","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.clinthera.2005.08.004_bib44","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1023\/A:1010387522195","article-title":"Rare diseases and the assessment of intervention: What sorts of clinical trials can we use?","volume":"24","author":"Wilcken","year":"2001","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.clinthera.2005.08.004_bib45","series-title":"Guideline on clinical trials in small populations","author":"European Medicines Agency, Committee for Medicinal Products for Human Use (CHMP)","year":"2005"}],"container-title":["Clinical Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0149291805001529?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0149291805001529?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2021,7,18]],"date-time":"2021-07-18T05:31:46Z","timestamp":1626586306000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0149291805001529"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2005,8]]},"references-count":45,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2005,8]]}},"alternative-id":["S0149291805001529"],"URL":"https:\/\/doi.org\/10.1016\/j.clinthera.2005.08.004","relation":{},"ISSN":["0149-2918"],"issn-type":[{"value":"0149-2918","type":"print"}],"subject":[],"published":{"date-parts":[[2005,8]]}}}