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Acta Paediatrica, Volume 94 Supplement 447 2005

ISSN: 1651-2227 (electronic) 0803-5253 (paper)
Publication Frequency: 12 issues per year

The circumstances under which this title is published have changed:

Reason for change: Changed Publisher
Now published by: Blackwell
Date of change: 31 December 2006

    Issue Purchase: No online purchase options available.
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Preface
Foreword
Michael Beck;  Timothy M. Cox;  Atul Mehta; Urs Widmer
Pages 5 – 6
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Commentary
Commentary
U. Widmer
Pages 9 – 10
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REVIEW
Fabry disease and the heart: an overview of the natural history and the effect of enzyme replacement therapy
J. S. Shah; P. M. Elliott
Pages 11 – 14
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ORIGINAL ARTICLE
The right ventricle in Fabry disease
C. Kampmann;  F. A. Baehner;  C. Whybra;  M. Bajbouj;  K. Baron;  M. Knuf;  C. M. Wiethoff;  H. Trübel; M. Beck
Pages 15 – 18
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REVIEW
Measurement of renal function in patients with Fabry disease
J. Kleinert;  M. Lorenz;  A. -C. Hauser;  A. Becherer;  A. Staudenherz;  M. Födinger; G. Sunder-Plassmann
Pages 19 – 23
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Natural history of the cerebrovascular complications of Fabry disease
A. Mehta; L. Ginsberg
Pages 24 – 27
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Vascular complications of Fabry disease: enzyme replacement and other therapies
D. A. Hughes; A. B. Mehta
Pages 28 – 33
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Commentary
Commentary
T. M. Cox; B. Winchester
Pages 37 – 38
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REVIEW
Biomarkers in lysosomal storage diseases: a review
T. M. Cox
Pages 39 – 42
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Identification and use of biomarkers in Gaucher disease and other lysosomal storage diseases
J. M. F. G. Aerts;  C. E. M. Hollak;  M. van Breemen;  M. Maas;  J. E. M. Groener; R. G. Boot
Pages 43 – 46
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Clinical evaluation of biomarkers in Gaucher disease
P. B. Deegan; T. M. Cox
Pages 47 – 50
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ORIGINAL ARTICLE
Is globotriaosylceramide a useful biomarker in Fabry disease?
E. Young;  K. Mills;  P. Morris;  A. Vellodi;  P. Lee;  S. Waldek; B. Winchester
Pages 51 – 54
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Commentary
Commentary
M. Beck; G. Andria
Page 57
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REVIEW
Enzyme replacement therapy in mucopolysaccharidosis type I
E. Miebach
Pages 58 – 60
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ORIGINAL ARTICLE
Pharmacokinetic profile of recombinant human N-acetylgalactosamine 4-sulphatase enzyme replacement therapy in patients with mucopolysaccharidosis VI (Maroteaux–Lamy syndrome): a phase I/II study
P. Harmatz;  W. G. Kramer;  J. J. Hopwood;  J. Simon;  E. Butensky; S. J. Swiedler
Pages 61 – 68
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REVIEW
Substrate reduction therapy for lysosomal storage diseases
T. M. Cox
Pages 69 – 75
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COMMENTARY

A. Mehta; J. -J. Cassiman
Page 79
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ORIGINAL ARTICLE
What can cell biology tell us about heterogeneity in lysosomal storage diseases?
V. Gieselmann
Pages 80 – 86
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Genotype and phenotype in Fabry disease: analysis of the Fabry Outcome Survey
E. Schaefer;  A. Mehta; A. Gal
Pages 87 – 92
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Abstracts
Poster abstracts
Pages 95 – 133
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