Treatment with agalsidase beta during pregnancy in Fabry disease.
Fabry disease is an X-linked lysosomal storage disease caused by a deficiency of alpha-galactosidase A, which leads to excessive accumulation of glycosphingolipids in most tissues in the body, with life-threatening clinical consequences in the kidney, heart, and cerebrovascular system. Enzyme replacement therapy using exogenously produced alpha-galactosidase has been available for treatment of this multisystem progressive disease since 2001. Two different preparations of enzyme replacement therapy for Fabry disease are available outside of the USA: agalsidase alfa and agalsidase beta. Despite being X-linked, Fabry disease affects many female patients, and this report presents a successful pregnancy of a female patient receiving agalsidase beta.
Politei
Juan M
JM
Neurology and Lysosomal Disorders Service, Juan A. Fernandez Hospital, Buenos Aires, Argentina. jpolitei@hotmail.com
D056947:Enzyme Replacement Therapy; D000795:Fabry Disease / Q000188:drug therapy*; D005260:Female; D006801:Humans; D007527:Isoenzymes / Q000627:therapeutic use*; D011247:Pregnancy; D011256:Pregnancy Outcome; D016896:Treatment Outcome; D000519:alpha-Galactosidase / Q000627:therapeutic use*
D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta