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在“Elsevier电子期刊”中，命中：52条，耗时：小于0.01 秒

在所有数据库中总计命中：20条

1.Burden of Fabry disease in young patients (≤ 30 years of age) who were initiated on enzyme replacement therapy with agalsidase beta: a Fabry Registry analysis

作者：Robert J. Hopkin^a ; Eva Brand^b ; Ulla F. Rasmussen^c ; Dominique P. Germain^d ; Nathalie Guffon^e ; Ana Jovanovic^f ; Ilkka Kantola^g ; Amel Karaa^h ; Roberta Lemayⁱ ; Ana M. Martins^j ; William R. Wilcox^k ; Han-Wook Yoo^l

刊名：Molecular Genetics and Metabolism

出版年：2017

2.A randomized, phase 3B, open-label, parallel-group study of agalsidase beta in treatment-naive male pediatric patients with Fabry disease without severe symptoms (FIELD study): GL-3 clearance from superficial skin capillary endothelium

作者：Uma Ramaswami^a ; Frits A Wijburg^b ; Daniel G Bichet^c ; Lorne A Clarke^d ; Gabriela Dostalova^e ; Alejandro Fainboim^f ; Andreas Fellgiebel^g ; Cassiano Forcelini^h ; Kristina An Haackⁱ ; Robert J Hopkin^j ; Michael Mauer^k ; Behzad Najafian^k ; C. Ronald Scott^l ; Suma P Shankar^m ; Beth L Thurbergⁿ ; Camilla Tø ; ndel^o ; Anna Tylki-Szymanska^p ; Bernard Bé ; nichou^q

刊名：Molecular Genetics and Metabolism

出版年：2017

3.A randomized, phase 3B, open-label, parallel-group study of agalsidase beta in treatment-naive male pediatric patients with Fabry disease without severe symptoms (FIELD study): GL-3 clearance from kidney cells

刊名：Molecular Genetics and Metabolism

出版年：2017

4.Differences in cleavage of globotriaosylceramide and its derivatives accumulated in organs of young Fabry mice following enzyme replacement therapy

作者：Takashi Kodama^a ; Takahiro Tsukimura^a ; Ikuo Kawashima^b ; Atsuko Sato^c ; Hitoshi Sakuraba^c ; Tadayasu Togawa^a ; ^{tadayasu@my-pharm.ac.jp}

关键词：Fabry disease ; α-Galactosidase A ; α-Galactosidase A knockout mouse ; Globotriaosylceramide ; Globotriaosylsphingosine ; Enzyme replacement therapy

刊名：Molecular Genetics and Metabolism

出版年：2017

5.Improvement of Fabry disease pain with calcium gluconate infusions given at time of agalsidase beta therapy

刊名：Molecular Genetics and Metabolism

出版年：2017

6.Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry

关键词：eGFR ; estimated glomerular filtration rate ; ERT ; enzyme replacement therapy ; ESRD ; end-stage renal disease ; GL-3 ; globotriaosylceramide ; IEC ; independent ethics committee ; IRB ; institutional review board ; LVH ; left ventricular hypertrophy ; UP/Cr ; urinary protein/creatinine ratio

刊名：Molecular Genetics and Metabolism

出版年：2016

7.Comparison of integrated white blood cell alpha-galactosidase a activity exposure between every-other-day orally administered migalastat and biweekly infusions of agalsidase beta or agalsidase alfa

作者：Franklin K. Johnson ; Kenneth Valenzano ; Jeffrey Castelli

刊名：Molecular Genetics and Metabolism

出版年：2016

8.Anti-proteinuric therapy and Fabry nephropathy; factors associated with preserved kidney function during agalsidase-beta therapy

作者：David G. Warnock^a ; Christie P. Thomas^b ; Bojan Vujkovac^c ; Ruth C. Campbell^d ; Joel Charrow^e ; Dawn A. Laney^f ; Leslie L. Jackson^a ; William R. Wilcox^f ; Christoph Wanner^g

刊名：Molecular Genetics and Metabolism

出版年：2016

9.Improvement of Fabry disease related gastrointestinal symptoms in a significant proportion of female patients treated with agalsidase beta

作者：Robert Hopkin^a ; Ulla Feldt-Rasmussen^b ; Ana Maria Martins^c ; Alberto Ortiz^d ; Frank Weidemann^e ; Roberta Lemay^f ; William R. Wilcox^g

刊名：Molecular Genetics and Metabolism

出版年：2016

10.Agalsidase beta and infusion-related reactions: Findings in Brazilian patients

作者：Marco A. Curiati^a ; Carmen Silvia C. Mendes^a ; Carolina S. Aranda^a ; Murilo Maia^b ; Luis Felipe Ensina^a ; Dirceu Solé ; ^a ; Ana Maria Martins^a

刊名：Molecular Genetics and Metabolism

出版年：2016

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