𝔖 Bobbio Scriptorium
✦   LIBER   ✦

Swiss national guideline for reimbursement of enzyme replacement therapy in late-onset Pompe disease

✍ Scribed by Hundsberger, Thomas; Rohrbach, Marianne; Kern, Lukas; Rösler, Kai M.


Book ID
120734023
Publisher
Springer
Year
2013
Tongue
English
Weight
663 KB
Volume
260
Category
Article
ISSN
0340-5354

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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