Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study

BACKGROUND: Pompe disease is a rare metabolic myopathy for which disease-specific enzyme replacement therapy (ERT) has been available since 2006. ERT has shown efficacy concerning muscle strength and pulmonary function in adult patients. However, no data on the effect of ERT on the survival of adult...

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Bibliografische gegevens
Hoofdauteurs: Güngör, Deniz, Kruijshaar, Michelle E, Plug, Iris, D’Agostino, Ralph B, Hagemans, Marloes LC, van Doorn, Pieter A, Reuser, Arnold JJ, van der Ploeg, Ans T
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: BioMed Central 2013
Onderwerpen:
Research
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3623847/
https://ncbi.nlm.nih.gov/pubmed/23531252
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-8-49
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