Pediatrics: implementing the promise of early intervention for Fabry disease.
Raas-Rothschild
Annick
A
Department of Human Genetics, Hadassah-Hebrew University Hospital, Jerusalem 91120. annick@hadassah.org.il
D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D000795:Fabry Disease / Q000175:diagnosis / Q000188:drug therapy / Q000235:genetics; D005260:Female; D006801:Humans; D007527:Isoenzymes / Q000627:therapeutic use*; D008297:Male; D012042:Registries; D000519:alpha-Galactosidase / Q000627:therapeutic use*
D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta