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

Deniz Gungor, Michelle Kruijshaar, I (Iris) Plug, RB D'Agostino, Marloes Hagemans, Pieter van Doorn, Arnold Reuser, Ans van der Ploeg

Research output: Contribution to journalArticleAcademicpeer-review

71 Citations (Scopus)
1 Downloads (Pure)
Original languageUndefined/Unknown
JournalOrphanet Journal of Rare Diseases
Publication statusPublished - 2013

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