Enzyme replacement therapy in severe Fabry disease with renal failure: a 1-year follow-up
DOI:
https://doi.org/10.1080/00015550410030682Abstract
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.Downloads
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