Agalsidase alfa: specific treatment for Fabry disease.
Fabry disease is a rare genetic lysosomal storage disorder characterized by a deficiency of the enzyme alpha-galactosidase A. The recent availability of enzyme-replacement therapy with agalsidase alfa offers specific treatment for this serious, progressive condition.
Mehta
Atul
A
Royal Free Hospital, London NW3 2QG.
D000368:Aged; D016527:Drug Costs; D000795:Fabry Disease / Q000188:drug therapy* / Q000191:economics / Q000235:genetics; D005260:Female; D006801:Humans; D007527:Isoenzymes / Q000172:deficiency / Q000191:economics / Q000627:therapeutic use*; D008297:Male; D011994:Recombinant Proteins; D016896:Treatment Outcome; D000519:alpha-Galactosidase / Q000191:economics / Q000627:therapeutic use*
D007527:Isoenzymes; D011994:Recombinant Proteins; C000627036:agalsidase alfa; D000519:alpha-Galactosidase