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

被引：18

作者：

Wanner, Christoph ^{[1
]}

Gerrnain, Dominique P. ^{[2
,3
]}

Hilz, Max J. ^{[4
]}

Spada, Marco ^{[5
]}

Falissard, Bruno ^{[6
]}

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

机构：

[1] Univ Wurzburg, Univ Clin, Div Nephrol, Oberdurrbacherstr 6, D-97080 Wurzburg, Germany

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

[3] Paris Saclay Univ, Univ Versailles, INSEAM U1179, Montigny, France

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

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

[6] Univ Paris 05, Univ Paris Sud, INSERM U1018, Paris, France

[7] UCL, London, England

[8] Barts Heart Ctr, London, England

来源：

MOLECULAR GENETICS AND METABOLISM | 2019年 / 126卷 / 03期

关键词：

D O I：

10.1016/j.ymgme.2018.04.004

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：210 / 211

页数：2

共 4 条

[1]

[Anonymous], 2018, MOL GENET METAB

[2]

Elliott P. M., 2018, MOL GENET METAB

[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].

Germain, Dominique P. ;

Elliott, Perry M. ;

Falissard, Bruno ;

Fomin, Victor V. ;

Hilz, Max J. ;

Jovanovic, Ana ;

Kantola, Ilkka ;

Linhart, Ales ;

Mignani, Renzo ;

Namdar, Mehdi ;

Nowak, Albina ;

Oliveira, Joao-Paulo ;

Pieroni, Maurizio ;

Viana-Baptista, Miguel ;

Wanner, Christoph ;

Spada, Marco .

MOLECULAR GENETICS AND METABOLISM REPORTS, 2019, 19

[4]

Wanner C., 2018, MOL GENET METAB

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