European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience-Reference-Cited by-同舟云学术

European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience

Published:2017-05-06 Issue:6 Volume:24 Page:768-e31
ISSN:1351-5101
Container-title:European Journal of Neurology
language:en
Short-container-title:Eur J Neurol

Author:

van der Ploeg A. T.¹^ORCID,Kruijshaar M. E.¹,Toscano A.²,Laforêt P.³,Angelini C.⁴,Lachmann R. H.⁵,Pascual Pascual S. I.⁶⁷,Roberts M.⁸,Rösler K.⁹,Stulnig T.¹⁰,van Doorn P. A.¹¹,Van den Bergh P. Y. K.¹²,Vissing J.¹³,Schoser B.¹⁴,

Affiliation:

1. Center for Lysosomal and Metabolic Diseases; Erasmus MC University Medical Center; Rotterdam the Netherlands

2. Department of Clinical and Experimental Medicine; University of Messina; Messina Italy

3. Paris-Est; Referral Center for Neuromuscular Disorders; Pitié-Salpêtrière Hospital; Assistance Publique Hôpitaux de Paris; Paris France

4. Fondazione S. Camillo Hospital; IRCCS; Venice Italy

5. Charles Dent Metabolic Unit; National Hospital for Neurology and Neurosurgery; London UK

6. Servicio de Neuropediatria; Hospital Universitario; La Paz Madrid

7. Department of Paediatrics; Universidad Autónoma de Madrid; Madrid Spain

8. Department of Neurology; Salford Royal NHS Foundation Trust; Salford UK

9. Neuromuscular Centre; University Department of Neurology; Inselspital; Bern Switzerland

10. Department of Medicine III; Medical University of Vienna; Vienna Austria

11. Department of Neurology; Erasmus MC University Medical Center; Rotterdam the Netherlands

12. Department of Neurology; Neuromuscular Reference Centre; University Hospitals St-Luc; Brussels Belgium

13. Copenhagen Neuromuscular Center; Rigshospitalet; University of Copenhagen; Copenhagen Denmark

14. Friedrich-Baur-Institute; Ludwig-Maximilians-University; Munich Germany

Funder

European Neuromuscular Centre

Publisher

Wiley

Subject

Neurology (clinical),Neurology

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1111/ene.13285/fullpdf

Reference75 articles.

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3. Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial;Amalfitano;Genet Med,2001

4. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease;Kishnani;Neurology,2007

5. Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease;Kishnani;J Pediatr,2006

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