Enzyme replacement therapy in severe Fabry disease with renal failure: a 1-year follow-up

Acta Derm Venereol. 2004;84(5):389-92. doi: 10.1080/00015550410030682.

Abstract

We present here the course of clinical response of a 53-year-old haemodialysed Fabry patient who received recombinant human alpha-galactosidase A at a dose of 1 mg/kg every other week over a period of 1 year. The therapy was well tolerated by the patient, who revealed an impressive favourable cutaneous, gastrointestinal, neurological and psychiatric response and a dramatic improvement in his quality of life, but no improvement in cardiac and renal function.

Publication types

  • Case Reports

MeSH terms

  • Enzyme Therapy*
  • Fabry Disease / complications
  • Fabry Disease / drug therapy*
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Renal Dialysis
  • Renal Insufficiency / etiology*
  • Renal Insufficiency / therapy
  • Treatment Outcome
  • alpha-Galactosidase / therapeutic use*

Substances

  • alpha-Galactosidase