Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study

BACKGROUND: Due partly to physicians’ unawareness, many adults with Pompe disease are diagnosed with great delay. Besides, it is not well known which factors influence the rate of disease progression, and thus disease outcome. We delineated the specific clinical features of Pompe disease in adults,...

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Autors principals: van der Beek, Nadine AME, de Vries, Juna M, Hagemans, Marloes LC, Hop, Wim CJ, Kroos, Marian A, Wokke, John HJ, de Visser, Marianne, van Engelen, Baziel GM, Kuks, Jan BM, van der Kooi, Anneke J, Notermans, Nicolette C, Faber, Karin G, Verschuuren, Jan JGM, Reuser, Arnold JJ, van der Ploeg, Ans T, van Doorn, Pieter A
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2012
Matèries:
Research
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3551719/
https://ncbi.nlm.nih.gov/pubmed/23147228
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-7-88
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