Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

BACKGROUND: Pompe disease is a rare lysosomal storage disorder characterized by muscle weakness and wasting. The majority of adult patients have slowly progressive disease, which gradually impairs mobility and respiratory function and may lead to wheelchair and ventilator dependency. It is as yet un...

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Библиографические подробности
Главные авторы: Güngör, Deniz, de Vries, Juna M, Hop, Wim CJ, Reuser, Arnold JJ, van Doorn, Pieter A, van der Ploeg, Ans T, Hagemans, Marloes LC
Формат: Artigo
Язык:Inglês
Опубликовано: BioMed Central 2011
Предметы:
Research
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3135500/
https://ncbi.nlm.nih.gov/pubmed/21631931
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-6-34
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