Burden associated with Fabry disease and its treatment in 12–15 year olds: results from a European survey
Abstract 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...
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| Principais autores: | , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
BMC
2022-07-01
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| Serier: | Orphanet Journal of Rare Diseases |
| Fag: | |
| Online adgang: | https://doi.org/10.1186/s13023-022-02417-3 |
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