About: A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I

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scientific article published on 26 November 2008

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rdf:type	Item
description	wetenschappelijk artikel (nl) article scientifique publié en 2008 (fr) наукова стаття, опублікована в листопаді 2008 (uk) im November 2008 veröffentlichter wissenschaftlicher Artikel (de) scientific article published on 26 November 2008 (en) artículu científicu espublizáu en payares de 2008 (ast)
publication date	wds:Q82749809-4DF38DCE-1C95-4C6C-81D4-8EA15DEB2BBF
publication date	2008-11-26 00:00:00Z (xsd:dateTime)
cites work	wds:Q82749809-0DA38DC1-03CD-4AAD-B59D-9DA57FBDC946 wds:Q82749809-11C381D3-8D97-481C-98E4-4E6B931B1A4E wds:Q82749809-1BAE24DF-F5C8-4103-8CA9-C29C11B702B5 wds:Q82749809-31B5B6E9-B3A3-41CF-8F51-4A77B107A1E0 wds:Q82749809-5C77807D-546A-41F4-A97E-16426AD7C823 wds:Q82749809-6B225927-9FB8-4973-AACB-48809D722013 wds:Q82749809-6D38FD29-5F73-42BF-AB23-3C4296CED6B9 wds:Q82749809-6F8E37E2-92EF-4C0B-A5F8-73F32F763A2F wds:Q82749809-8A04C6BC-B586-4D2E-8820-FAC24B79863B wds:Q82749809-B297D87C-3422-4DA2-A6BE-FCE74DDD1292 wds:Q82749809-B32B4391-C2FD-4772-AD60-B95F0B5DDE13 wds:Q82749809-D7ECD475-42EB-4B98-A7EC-5E6DA2792258 wds:Q82749809-D919DF38-7DFF-49EF-A4AB-43969674D810 wds:Q82749809-FEE6BFFA-2393-411F-85D9-9872C4739285
cites work	Incidence of inborn errors of metabolism in British Columbia, 1969-1996. Interpreting small differences in functional status: the Six Minute Walk test in chronic lung disease patients Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. The frequency of lysosomal storage diseases in The Netherlands. Prevalence of lysosomal storage disorders The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). Enzyme replacement therapy for the mucopolysaccharide storage disorders A review and selection of simple laboratory methods used for the study of glycosaminoglycan excretion and the diagnosis of the mucopolysaccharidoses Enzyme-replacement therapy in mucopolysaccharidosis I. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase). Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells The clinical spectrum of alpha-L-iduronidase deficiency Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.
author name string	wds:Q82749809-3A4282AE-222E-4F1C-AB1B-81B0C8C43526 wds:Q82749809-4F971EAF-10D9-4827-8895-45A09B45EA43 wds:Q82749809-78924178-532D-421C-990C-955A8E43A28C wds:Q82749809-869C5BEE-345A-46CF-AD69-E1D584C367FC wds:Q82749809-8F13471A-AD16-4DA6-86C1-F1C277E6EEBD wds:Q82749809-958B857E-D375-41AE-9A1E-67337B8DDE49 wds:Q82749809-D0E9452C-B0AA-478E-B3A4-8A201EF2C3EF wds:Q82749809-FA0A277C-144A-46E2-8282-B094B2BCAE03 wds:Q82749809-FE27291F-51BA-4494-A061-680BC1BCCBE0
author name string	Gerald F Cox Ana Maria Martins Emil D Kakkis Joe T R Clarke Eugênia R Valadares Marisa Sidman José E C Góes Mary Alice Worden Verónica Muñoz Rojas
rdfs:label	A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I (en) A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I (nl)
skos:prefLabel	A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I (en) A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I (nl)
name	A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I (en) A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I (nl)
author	wds:Q82749809-410C8968-54C3-4014-A3EB-F27C7231EED5
author	Roberto Giugliani
title	wds:Q82749809-2E3DBACF-6523-449F-B80C-8535AE324E32
title	A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I (en)
page(s)	wds:Q82749809-6227D506-BA0E-43F1-B2F5-C5F73B8C74AD
page(s)	13-19
instance of	wds:Q82749809-0B8FE309-8C68-4DB1-848D-A109BA1E76F2
instance of	scholarly article
main subject	wds:Q82749809-4D1D186B-21D4-4F48-8060-33E9543AA848 wds:Q82749809-565FEB7D-8CCC-429A-91FD-B64EB156EFA2
main subject	mucopolysaccharidosis I laronidase
PubMed ID	wds:Q82749809-BA4F9A22-B721-46D1-8DA2-ECF150CB79E1
PubMed ID	http://rdf.ncbi.nlm.nih.gov/pubchem/reference/19038563
PubMed ID	19038563
published in	wds:Q82749809-6D13077D-6363-4A46-935C-EE0990511585
published in	Molecular Genetics and Metabolism
issue	wds:Q82749809-0F2079DA-87E9-4820-A006-52040D1EF1B3
volume	wds:Q82749809-22C0D1CD-35D7-4BDA-8B95-28DE248F5A37
issue	1
volume	96
DOI	wds:Q82749809-9DE79901-C850-46A9-901B-98CA2043293D
DOI	http://dx.doi.org/10.1016/J.YMGME.2008.10.009
DOI	10.1016/J.YMGME.2008.10.009
is about of	https://www.wikidata.org/wiki/Special:EntityData/Q82749809
is cites work of	Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration Childhood onset of Scheie syndrome, the attenuated form of mucopolysaccharidosis I. Efficient analysis of urinary glycosaminoglycans by LC-MS/MS in mucopolysaccharidoses type I, II and VI A pilot newborn screening program for Mucopolysaccharidosis type I in Taiwan Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long-term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans. Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction. Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment. Longitudinal observations of serum heparin cofactor II-thrombin complex in treated Mucopolysaccharidosis I and II patients Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy Effects of enzyme replacement therapy started late in a murine model of mucopolysaccharidosis type I Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis. Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 t

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