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Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry
BACKGROUND: Agalsidase β is a form of enzyme replacement therapy for Fabry disease, a genetic disorder characterised by low α-galactosidase A activity, accumulation of glycosphingolipids and life-threatening cardiovascular, renal and cerebrovascular events. In clinical trials, agalsidase β cleared g...
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| Publicado en: | J Med Genet |
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| Autores principales: | , , , , , , , , , , , , , , , , , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
BMJ Publishing Group
2016
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| Materias: | |
| Acceso en línea: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4941144/ https://ncbi.nlm.nih.gov/pubmed/26993266 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1136/jmedgenet-2015-103486 |
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