Recombinant enzyme replacement therapy for infantile-onset Pompe disease
โ Scribed by Michio Hirano
- Book ID
- 107545571
- Publisher
- Springer
- Year
- 2008
- Tongue
- English
- Weight
- 96 KB
- Volume
- 8
- Category
- Article
- ISSN
- 1528-4042
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๐ SIMILAR VOLUMES
## Abstract ## Introduction: Enzyme replacement therapy (ERT) in ultraโorphan Pompe disease generates antiโrhGAA antibodies, which may interfere with efficacy. ## Methods: rhGAAโspecific Tโcell responses were examined at different timeโpoints in 6 Hungarian patients treated with rhGAA and compar
## Abstract Pompe's disease (glycogen storage disease type II) is an autosomal recessive myopathy caused by lysosomal ฮฑโglucosidase deficiency. Enzyme replacement therapy (ERT) is currently under development for this disease. We evaluated the morphological changes in muscle tissue of four children