About: Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey     Goto   Sponge   NotDistinct   Permalink

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  • 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)
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)
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)
skos:notation
  • RIV/00064165:_____/09:5781!RIV10-MZ0-00064165
http://linked.open...avai/riv/aktivita
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  • N
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  • 8
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  • 328612
http://linked.open...ai/riv/idVysledku
  • RIV/00064165:_____/09:5781
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  • enzyme replacement therapy; agalsidase-alpha; clinical-manifestations; imvolvement; prevalence; efficacy; galactosidase (en)
http://linked.open.../riv/klicoveSlovo
http://linked.open...odStatuVydavatele
  • GB - Spojené království Velké Británie a Severního Irska
http://linked.open...ontrolniKodProRIV
  • [1DA4BC037B80]
http://linked.open...i/riv/nazevZdroje
  • Journal of Medical Genetics
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  • 46
http://linked.open...iv/tvurceVysledku
  • Beck, M.
  • Linhart, Aleš
  • Mehta, A.
  • Giugliani, R.
  • Sunder-Plassmann, G.
  • Clarke, J T R
  • Elliott, P.
http://linked.open...ain/vavai/riv/wos
  • 000268541500006
issn
  • 0022-2593
number of pages
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