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Burden associated with Fabry disease and its treatment in 12–15 year olds: results from a European survey
BACKGROUND: Fabry Disease (FD) is a rare X-linked metabolic lysosomal disorder. FD has a broad range of symptoms which vary markedly between patients. The heterogenous nature of the disease makes diagnosis difficult for health care professionals (HCPs), which in turn has a significant effect on the...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9287883/ https://www.ncbi.nlm.nih.gov/pubmed/35840992 http://dx.doi.org/10.1186/s13023-022-02417-3 |