lunes, 1 de octubre de 2018

Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact? | Orphanet Journal of Rare Diseases | Full Text

Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact? | Orphanet Journal of Rare Diseases | Full Text

Orphanet Journal of Rare Diseases

Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?

Orphanet Journal of Rare Diseases201813:171
  • Received: 27 August 2018
  • Accepted: 21 September 2018
  • Published: 

Abstract

Fabry disease (FD) is a rare X-linked disorder caused by a deficiency of lysosomal α-galactosidase A activity. Treatment with recombinant enzyme replacement therapy is available since 2001 and the effects of anti-drug antibodies (ADA) on therapy efficacy and disease outcome in affected patients have been controversially reported. In this letter we discuss the importance of adequate measurements of neutralizing ADAs and appropriate longitudinal analysis to determine therapy efficiency and clinical outcome in patients with FD.

Keywords

  • Enzyme replacement therapy
  • Longitudinal
  • Prospective

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