Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry
Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry
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Publisher
Berlin/Heidelberg: Springer-Verlag
Journal title
Language
English
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Publication information
Publisher
Berlin/Heidelberg: Springer-Verlag
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Scope and Contents
Contents
Our objective was to assess how the diagnosis and treatment of mucopolysaccharidosis I (MPS I) have changed over time. We used data from 891 patients in the MPS I Registry, an international observational database, to analyze ages at symptom onset, diagnosis, treatment initiation, and treatment allocation (hematopoietic stem cell transplantation, en...
Alternative Titles
Full title
Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry
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Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3357468
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3357468
Other Identifiers
ISSN
0340-6199
E-ISSN
1432-1076
DOI
10.1007/s00431-011-1644-x