Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy-Reference-Cited by-同舟云学术

Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy

Published:2018-03-19 Issue:6 Volume:41 Page:1205-1214
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

van der Meijden Jan C.¹,Kruijshaar Michelle E.¹,Harlaar Laurike²,Rizopoulos Dimitris³,van der Beek Nadine A. M. E.²,van der Ploeg Ans T.¹^ORCID

Affiliation:

1. ; Center for Lysosomal and Metabolic Diseases, Department of Pediatrics; Erasmus MC University Medical Center; Wytemaweg 80 Rotterdam 3015 CN The Netherlands

2. ; Center for Lysosomal and Metabolic Diseases, Department of Neurology; Erasmus MC University Medical Center; Rotterdam the Netherlands

3. ; Department of Biostatistics; Erasmus MC University Medical Center; Rotterdam the Netherlands

Funder

University Medical Center Rotterdam

ZonMW - the Netherlands Organization for Health Research and Development

Prinses Beatrix Fonds

TKI - Health Holland

Tex Net, SSWO

Metakids

“Conselho Nacional de Desenvolvimento Científico e Tecnológico” - the National Counsel of Technological and Scientific Development, Brasil

Colciencias and Sanofi Genzyme

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://link.springer.com/article/10.1007/s10545-018-0166-3/fulltext.html

Reference53 articles.

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2. ATS/ERS statement on respiratory muscle testing;American Thoracic Society/European Respiratory Society;Am J Respir Crit Care Med,2002

3. Effectiveness of enzyme replacement therapy in adults with late-onset Pompe disease: results from the NCS-LSD cohort study;Anderson;J Inherit Metab Dis,2014

4. Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years;Angelini;J Neurol,2012

5. The influence of a polymorphism in the gene encoding angiotensin converting enzyme (ACE) on treatment outcomes in late-onset Pompe patients receiving alglucosidase alfa;Baek;Mol Genet Metab Rep,2016

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4. Inborn Errors of Carbohydrate, Ammonia, Amino Acid, and Organic Acid Metabolism;Avery's Diseases of the Newborn;2024

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