Long-term safety and effectiveness of velaglucerase alfa in Gaucher disease: 6-year interim analysis of a post-marketing surveillance in Japan
Abstract Background Gaucher disease (GD) is caused by reduced lysosomal enzyme β-glucocerebrosidase activity. Heterogeneous genotypes and phenotypes have been observed within GD types and across ethnicities. Enzyme replacement therapy is generally recommended for patients with type 1 GD, the least s...
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| Auteurs principaux: | , , , , , |
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| Format: | article |
| Langue: | EN |
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BMC
2021
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| Sujets: | |
| Accès en ligne: | https://doaj.org/article/8d337695556a429a94e942a7534676ae |
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