Search Results - "Broekgaarden, H"

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    Ten years of the international Pompe survey: patient reported outcomes as a reliable tool for studying treated and untreated children and adults with non-classic Pompe disease by van der Meijden, J. C., Güngör, D., Kruijshaar, M. E., Muir, A. D. J., Broekgaarden, H. A., van der Ploeg, A. T.

    Published in Journal of inherited metabolic disease (01-05-2015)
    “…Pompe disease is a rare, progressive lysosomal storage disorder for which enzyme therapy (ERT) became available in 2006. Four years earlier, the IPA/Erasmus MC…”
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    Journal Article
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