208th ENMC International Workshop: Formation of a European Network to develop a European data sharing model and treatment guidelines for Pompe disease Naarden, The Netherlands, 26–28 September 2014-Reference-Cited by-同舟云学术

208th ENMC International Workshop: Formation of a European Network to develop a European data sharing model and treatment guidelines for Pompe disease Naarden, The Netherlands, 26–28 September 2014

Published:2015-08 Issue:8 Volume:25 Page:674-678
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Schoser Benedikt,Laforêt Pascal,Kruijshaar Michelle E.,Toscano Antonio,van Doorn Pieter A.,van der Ploeg Ans T.

Funder

European Neuromuscular Centre (ENMC)

Muskelsvindfonden (Denmark)

Association Française contre les Myopathies (France)

Deutsche Gesellschaft für Muskelkranke (Germany)

Telethon Foundation (Italy)

Schweizerische Stiftung für die Erforschung der Muskelkrankheiten (Switzerland)

Prinses Beatrix Fonds (The Netherlands)

Spierziekten Nederland (The Netherlands)

Muscular Dystrophy Campaign (UK)

Finnish Neuromuscular Association (Finland)

Dutch ZonMw

International Pompe Association (IPA)

Genzyme Europe BV

GlaxoSmithKline Rare Diseases

Amicus Therapeutics

Publisher

Elsevier BV

Subject

Genetics(clinical),Clinical Neurology,Neurology,Pediatrics, Perinatology, and Child Health

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3. Recombinant human alpha-glucosidase from rabbit milk in Pompe patients;Van den Hout;Lancet,2000

4. Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk;Van den Hout;Pediatrics,2004

5. Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study;de Vries;Orphanet J Rare Dis,2012

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