Enzyme Replacement Therapy for Pompe Disease
✍ Scribed by Corrado Angelini; Claudio Semplicini
- Book ID
- 107545881
- Publisher
- Springer
- Year
- 2011
- Tongue
- English
- Weight
- 297 KB
- Volume
- 12
- Category
- Article
- ISSN
- 1528-4042
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📜 SIMILAR VOLUMES
## 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
## ABSTRACT Fabry disease is a multisystem disorder associated with wide variability in clinical expression. Fabry disease is an X‐linked lysosomal storage disorder caused by a deficiency of α‐galactosidase A. The enzyme defect leads to the systemic accumulation of glycosphingolipids with α‐galacto