First two years of reimbursed enzyme replacement therapy in the treatment of Fabry disease in Poland [version 2; peer review: 2 approved]
Fabry disease (FD) is an ultra-rare genetic lysosomal storage disease caused by pathologic gene variants resulting in insufficient expression of α-galactosidase A. This enzyme deficiency leads to accumulation of globotriaosylceramide and globotriaosylsphingosine in plasma and in different cells thro...
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| Autores principales: | , , , , , , , , , |
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| Formato: | article |
| Lenguaje: | EN |
| Publicado: |
F1000 Research Ltd
2021
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| Materias: | |
| Acceso en línea: | https://doaj.org/article/8f9bce18977c489b93edd6aaf63201e9 |
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