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Twenty years of the Fabry Outcome Survey (FOS): insights, achievements, and lessons learned from a global patient registry

BACKGROUND: Patient registries provide long-term, real-world evidence that aids the understanding of the natural history and progression of disease, and the effects of treatment on large patient populations with rare diseases. The year 2021 marks the 20th anniversary of the Fabry Outcome Survey (FOS...

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Detalles Bibliográficos
Autores principales:	Beck, Michael, Ramaswami, Uma, Hernberg-Ståhl, Elizabeth, Hughes, Derralynn A., Kampmann, Christoph, Mehta, Atul B., Nicholls, Kathleen, Niu, Dau-Ming, Pintos-Morell, Guillem, Reisin, Ricardo, West, Michael L., Schenk, Jörn, Anagnostopoulou, Christina, Botha, Jaco, Giugliani, Roberto
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9208147/ https://www.ncbi.nlm.nih.gov/pubmed/35725623 http://dx.doi.org/10.1186/s13023-022-02392-9

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