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Description
| - Fabry disease is a rare X-linked lysosomal storage disorder characterised by severe multisystemic involvement that leads to major organ failure and premature death in affected men and women. Over the past 7 years, the Fabry Outcome Survey (FOS) has collected data on the natural history of Fabry disease, and the long-term efficacy and safety of enzyme-replacement therapy. This paper provides an update on the first analysis of FOS data.
- Fabry disease is a rare X-linked lysosomal storage disorder characterised by severe multisystemic involvement that leads to major organ failure and premature death in affected men and women. Over the past 7 years, the Fabry Outcome Survey (FOS) has collected data on the natural history of Fabry disease, and the long-term efficacy and safety of enzyme-replacement therapy. This paper provides an update on the first analysis of FOS data. (en)
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Title
| - Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey
- Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey (en)
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skos:prefLabel
| - Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey
- Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey (en)
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skos:notation
| - RIV/00064165:_____/09:5781!RIV10-MZ0-00064165
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http://linked.open...avai/riv/aktivita
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http://linked.open...avai/riv/aktivity
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http://linked.open...iv/cisloPeriodika
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http://linked.open...vai/riv/dodaniDat
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http://linked.open...aciTvurceVysledku
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http://linked.open.../riv/druhVysledku
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http://linked.open...iv/duvernostUdaju
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http://linked.open...titaPredkladatele
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http://linked.open...dnocenehoVysledku
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http://linked.open...ai/riv/idVysledku
| - RIV/00064165:_____/09:5781
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http://linked.open...riv/jazykVysledku
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http://linked.open.../riv/klicovaSlova
| - enzyme replacement therapy; agalsidase-alpha; clinical-manifestations; imvolvement; prevalence; efficacy; galactosidase (en)
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http://linked.open.../riv/klicoveSlovo
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http://linked.open...odStatuVydavatele
| - GB - Spojené království Velké Británie a Severního Irska
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http://linked.open...ontrolniKodProRIV
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http://linked.open...i/riv/nazevZdroje
| - Journal of Medical Genetics
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http://linked.open...in/vavai/riv/obor
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http://linked.open...ichTvurcuVysledku
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http://linked.open...cetTvurcuVysledku
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http://linked.open...UplatneniVysledku
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http://linked.open...v/svazekPeriodika
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http://linked.open...iv/tvurceVysledku
| - Beck, M.
- Linhart, Aleš
- Mehta, A.
- Giugliani, R.
- Sunder-Plassmann, G.
- Clarke, J T R
- Elliott, P.
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http://linked.open...ain/vavai/riv/wos
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issn
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number of pages
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