Fabry disease: the need to stratify patient populations to better understand the outcome of enzyme replacement therapy.
Germain
Dominique P
DP
Centre de référence de la maladie de Fabry et des maladies héréditaires du tissu conjonctif (syndromes d'Ehlers-Danlos et pseudoxanthome élastique), Assistance Publique-Hôpitaux de Paris, Paris, France. dominique.germain@egp.aphp.fr
D002986:Clinical Trials as Topic; D000795:Fabry Disease / Q000188:drug therapy*; D006801:Humans; D007527:Isoenzymes / Q000627:therapeutic use*; D010353:Patient Education as Topic; D016896:Treatment Outcome; D000519:alpha-Galactosidase / Q000502:physiology / Q000627:therapeutic use*
D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta