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Late onset Pompe disease revealed by newborn screening


Publisher
John Wiley and Sons
Year
2011
Tongue
English
Weight
154 KB
Volume
155
Category
Article
ISSN
1552-4825

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Enzyme replacement therapy induces T-cel
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## Abstract ## Introduction: Enzyme replacement therapy (ERT) in ultra‐orphan Pompe disease generates anti‐rhGAA antibodies, which may interfere with efficacy. ## Methods: rhGAA‐specific T‐cell responses were examined at different time‐points in 6 Hungarian patients treated with rhGAA and compar