Response to Gurevich and colleagues: The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: a systematic literature review by a European panel of experts

被引：1

作者：

Germain, Dominique P. ^{[1
,2
]}

Falissard, Bruno ^{[3
]}

Hilz, Max J. ^{[4
]}

Spada, Marco ^{[5
]}

Wanner, Christoph ^{[6
]}

Elliott, Perry M. ^{[7
,8
]}

机构：

[1] Paris Saclay Univ, Univ Versailles, Div Med Genet, French Referral Ctr Fabry Dis, Montigny, France

[2] Paris Saclay Univ, Univ Versailles, INSERM, U1179, Montigny, France

[3] Paris Descartes Univ, Univ Paris Sud, INSERM, U1018, Paris, France

[4] Univ Erlangen Nurnberg, Dept Neurol, Erlangen, Germany

[5] Univ Torino, Dept Paediat, Turin, Italy

[6] Univ Wurzburg, Univ Clin, Div Nephrol, Wurzburg, Germany

[7] UCL, London, England

[8] Barts Heart Ctr, London, England

来源：

MOLECULAR GENETICS AND METABOLISM REPORTS | 2019年 / 20卷

关键词：

Fabry disease; Enzyme replacement therapy; Agalsidase alfa; Agalsidase beta;

D O I：

10.1016/j.ymgmr.2019.100493

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

引用

页数：2

共 9 条

[1]

[Anonymous], 2009, Oxford centre for evidence-based medicine levels of evidence

[2] Why systematic literature reviews in Fabry disease should include all published evidence [J].

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[3] The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European panel of experts [J].

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Elliott, Perry M. ;

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[4] The effect of enzyme replacement therapy on clinical outcomes in female patients with Fabry disease - A systematic literature review by a European panel of experts [J].

Germain, Dominique P. ;

Arad, Michael ;

Burlina, Alessandro ;

Elliott, Perry M. ;

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[5] A response to Germain, et al. The effect of enzyme replacement therapy on clinical outcomes in male patients with Fabry disease: A systematic literature review by a European panel of experts. Mol Genet Metab Rep Feb 6 2019 [J].

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Schenk, Jorn ;

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[7] The effect of enzyme replacement therapy on clinical outcomes in paediatric patients with Fabry disease - A systematic literature review by a European panel of experts [J].

Spada, Marco ;

Baron, Ralf ;

Elliott, Perry M. ;

Falissard, Bruno ;

Hilz, Max J. ;

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[8] European expert consensus statement on therapeutic goals in Fabry disease [J].

Wanner, Christoph ;

Arad, Michael ;

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Fomin, Victor V. ;

Germain, Dominique P. ;

Hughes, Derralynn A. ;

Jovanovic, Ana ;

Kantola, Ilkka ;

Linhart, Ales ;

Mignani, Renzo ;

Monserrat, Lorenzo ;

Namdar, Mehdi ;

Nowak, Albina ;

Oliveira, Joao-Paulo ;

Ortiz, Alberto ;

Pieroni, Maurizio ;

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Hilz, Max J. .

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[9] Therapeutic goals in Fabry disease: Recommendations of a European expert panel, based on current clinical evidence with enzyme replacement therapy [J].

Wanner, Christoph ;

Gerrnain, Dominique P. ;

Hilz, Max J. ;

Spada, Marco ;

Falissard, Bruno ;

Elliott, Perry M. .

MOLECULAR GENETICS AND METABOLISM, 2019, 126 (03) :210-211

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