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...

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Detalles Bibliográficos
Autores principales: Bashorum, Lisa, McCaughey, Gerard, Evans, Owen, Humphries, Ashley C., Perry, Richard, MacCulloch, Alasdair
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
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

Internet

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

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