About: The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I

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scientific article published on 02 March 2007

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rdf:type	Item
description	wetenschappelijk artikel (nl) наукова стаття, опублікована в березні 2007 (uk) article scientifique publié en 2007 (fr) im März 2007 veröffentlichter wissenschaftlicher Artikel (de) artículu científicu espublizáu en marzu de 2007 (ast) scientific article published on 02 March 2007 (en) գիտական հոդված հրատարակված 2007 թվականի մարտի 2-ին (hy)
publication date	wds:Q79870081-7AD119D0-AED3-4D04-BAE0-CE1B1B181AAB
publication date	2007-03-02 00:00:00Z (xsd:dateTime)
cites work	wds:Q79870081-05D4A08C-8DC7-4D09-A6E6-32D123A90F32 wds:Q79870081-2FC1A7E3-C1BE-4004-B831-08A50889F7F0 wds:Q79870081-37670717-080B-48F6-9EC8-2ECBB5A04526 wds:Q79870081-4161456F-9C52-4E63-B6CE-F92D0454DCEA wds:Q79870081-4E1B6446-8F1F-40C6-A182-776E93944C6C wds:Q79870081-5FE095AC-0A62-4BE6-91CC-EBD02757D3E3 wds:Q79870081-64772162-29DA-45AE-B031-7D478AA112F1 wds:Q79870081-68775CF1-D559-4F58-8EBE-A8C1DF172455 wds:Q79870081-720AB840-EC45-47FD-AAC3-95718DFCEDAB wds:Q79870081-752CC476-D6F2-4F0C-BB08-CAA01CD4FDA9 wds:Q79870081-7C321437-DEAD-451D-85CA-E4F956CE31CC wds:Q79870081-7E1CC5C3-957F-47A2-9A52-03E6D3113AB2 wds:Q79870081-9327661D-DC1D-48CD-BE6D-34F02942B419 wds:Q79870081-A0DED131-D0C8-41E3-82BD-C7F130188F49 wds:Q79870081-A1526876-D6CB-40C6-9DE7-C1F0B08263EE wds:Q79870081-A4CF6257-7279-4E24-B793-377D01741500 wds:Q79870081-AF7759FB-AA15-4326-917B-C19843E472D4 wds:Q79870081-AF914711-796B-464A-BAE4-1E3F9040DCD4 wds:Q79870081-D215DA45-AF69-4924-A088-5EE2AF310193 wds:Q79870081-DBCD5777-09AA-4FB4-A3AB-63B10051B967 wds:Q79870081-E9834262-B852-427E-A303-FB2838B34FB6 wds:Q79870081-F32587A0-323B-4C58-91DB-6B1866AD8796 wds:Q79870081-F42ED09C-1C3A-4EFE-A0B6-51369D01DB89 wds:Q79870081-FAA3E021-7DA7-45BF-994A-79047B0BE438
cites work	Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources An update on the frequency of mucopolysaccharide syndromes in British Columbia The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease 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 Can mucopolysaccharidosis type I disease severity be predicted based on a patient's genotype? A comprehensive review of the literature Molecular genetics of mucopolysaccharidosis type I: diagnostic, clinical, and biological implications. The frequency of lysosomal storage diseases in The Netherlands. Incidence of the mucopolysaccharidoses in Northern Ireland Prevalence of lysosomal storage disorders Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I. Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: consensus recommendations Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). Cord-blood transplants from unrelated donors in patients with Hurler's syndrome Mutational analysis of 85 mucopolysaccharidosis type I families: frequency of known mutations, identification of 17 novel mutations and in vitro expression of missense mutations. Therapeutic goals in the treatment of Gaucher disease. Gaucher disease type 1: revised recommendations on evaluations and monitoring for adult patients Enzyme-replacement therapy in mucopolysaccharidosis I. The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Derivation and validation of a mathematical model for predicting the response to exogenous recombinant human growth hormone (GH) in prepubertal children with idiopathic GH deficiency. KIGS International Board. Kabi Pharmacia International Growth Stu Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres The mucopolysaccharidoses: a heterogeneous group of disorders with variable pediatric presentations Bridging regional and global perspectives Advances in growth hormone therapy: a new registry tool Problems in the use of large data sets to assess effectiveness
author name string	wds:Q79870081-2716BFBF-662D-44C9-BF36-9C90E3F9EB68 wds:Q79870081-3188B1B5-AD66-4194-9846-ABD6099C2656 wds:Q79870081-3EEF1C3E-F02E-400D-90C3-959C7BC21216 wds:Q79870081-45D97327-A8E5-472F-87B5-F2746F036641 wds:Q79870081-47002E86-D34D-4F33-8A58-30E6FC1098BA wds:Q79870081-4FB2AB1F-CD9E-4D55-A607-AAD477BC5411 wds:Q79870081-7636B609-666C-4B43-9AB7-7046BDDA6628 wds:Q79870081-7EEC2ED4-B74E-4752-88AB-C44D534E6E17 wds:Q79870081-97AE36A7-E4A5-471D-B14D-CBE8E2C22869 wds:Q79870081-BA9C5FB2-40AB-47C3-8058-4BFEC563B926 wds:Q79870081-C52EFC03-263B-4AD7-949F-699179CD7E01 wds:Q79870081-F4E33B94-1B51-44D1-8241-4B6330C60DFD
author name string	Gregory M Pastores Janet Thomas J Edmond Wraith Michael Beck Paige Kaplan Elsa Shapiro Pamela Arn David Viskochil Nathalie Guffon Joe T R Clarke Joseph Muenzer Denise Y J Norato
rdfs:label	The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I (en) The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I (nl)
skos:prefLabel	The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I (en) The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I (nl)
name	The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I (en) The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I (nl)
title	wds:Q79870081-B0A4CF5D-50C3-4C65-9C41-928F761E106C
title	The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I (en)
page(s)	wds:Q79870081-49ACBEE1-F62E-47E2-BC1C-16C1E0CE5A58
page(s)	37-47
instance of	wds:Q79870081-DB7B5965-BDDA-40E4-ADD6-C27B81EC12B4
instance of	scholarly article
main subject	wds:Q79870081-5D7671D3-C1B6-4BB6-AC58-0CE12C274718
main subject	mucopolysaccharidosis I
PubMed ID	wds:Q79870081-E5A0590B-757B-4973-8D8B-3DAF5C3C1ED6
PubMed ID	http://rdf.ncbi.nlm.nih.gov/pubchem/reference/17336562
PubMed ID	17336562
published in	wds:Q79870081-B44A94B3-0B9B-4430-A6AF-9B66101DAEDB

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