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

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...

Verfasser: Lenders, Malte
Schmitz, Boris
Brand, Stefan-Martin
Brand, Eva
FB/Einrichtung:FB 13: Biologie
Dokumenttypen:Artikel
Medientypen:Text
Erscheinungsdatum:2018
Publikation in MIAMI:25.07.2019
Datum der letzten Änderung:02.05.2022
Angaben zur Ausgabe:[Electronic ed.]
Quelle:Orphanet Journal of Rare Diseases 13 (2018 ) 171, 1-2
Schlagwörter:Enzyme replacement therapy; Longitudinal; Prospective
Fachgebiet (DDC):610: Medizin und Gesundheit
Lizenz:CC BY 4.0
Sprache:English
Förderung:Finanziert durch den Open-Access-Publikationsfonds 2018 der Deutschen Forschungsgemeinschaft (DFG) und der Westfälischen Wilhelms-Universität Münster (WWU Münster).
Format:PDF-Dokument
URN:urn:nbn:de:hbz:6-84129538247
Weitere Identifikatoren:DOI: 10.1186/s13023-018-0916-1
Permalink:https://nbn-resolving.de/urn:nbn:de:hbz:6-84129538247
Onlinezugriff:artikel_lenders_2018.pdf

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.