Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry.
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Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type IEnzyme replacement therapy with laronidase (Aldurazyme) for treating mucopolysaccharidosis type IEnzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type IEffects of enzyme replacement therapy started late in a murine model of mucopolysaccharidosis type IAminoglycoside-Induced Premature Stop Codon Read-Through of Mucopolysaccharidosis Type I Patient Q70X and W402X Mutations in Cultured Cells.The natural history of MPS I: global perspectives from the MPS I Registry.Anthropometric characteristics of four Polish children with mucopolysaccharidosis.Airway-related symptoms and surgeries in patients with mucopolysaccharidosis IMucopolysaccharidosis (MPS) Physical Symptom Score: Development, Reliability, and ValidityIdentification of infants at risk for developing Fabry, Pompe, or mucopolysaccharidosis-I from newborn blood spots by tandem mass spectrometry.The diagnostic journey of patients with mucopolysaccharidosis I: A real-world survey of patient and physician experiences.Immune tolerance induction for laronidase treatment in mucopolysaccharidosis I.Current and potential therapeutic strategies for mucopolysaccharidoses.Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series.Treatment of MPS I mice with microencapsulated cells overexpressing IDUA: effect of the prednisolone administration.Clinical and Molecular Characterization of Patients with Mucopolysaccharidosis Type I in an Algerian SeriesUnrelated CD3/CD19-depleted peripheral stem cell transplantation for Hurler syndrome.An algorithm to predict phenotypic severity in mucopolysaccharidosis type I in the first month of life.Social Functioning and Behaviour in Mucopolysaccharidosis IH [Hurlers Syndrome].SAAMP 2.0: an algorithm to predict genotype-phenotype correlation of lysosomal storage diseases.Failure to shorten the diagnostic delay in two ultra-orphan diseases (mucopolysaccharidosis types I and III): potential causes and implications.Mucopolysaccharidosis type I and craniosynostosis.A Unique Case of Cervical Myelopathy in an Adult Patient with Scheie Syndrome.Short stature as a presenting symptom of attenuated Mucopolysaccharidosis type I: case report and clinical insights
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P2860
Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry.
description
2012 nî lūn-bûn
@nan
2012 թուականի Յունուարին հրատարակուած գիտական յօդուած
@hyw
2012 թվականի հունվարին հրատարակված գիտական հոդված
@hy
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
name
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@ast
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@en
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@nl
type
label
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@ast
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@en
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@nl
prefLabel
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@ast
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@en
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@nl
P2093
P2860
P1476
Diagnosis and treatment trends ...... dings from the MPS I Registry.
@en
P2093
Frits Wijburg
Gerald F Cox
Kristin D'Aco
Lakshmi Rangachari
Lisa Underhill
Paige Kaplan
Pamela Arn
Roberto Giugliani
Torayuki Okuyama
P2860
P2888
P304
P356
10.1007/S00431-011-1644-X
P577
2012-01-11T00:00:00Z
P5875
P6179
1006402348