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...

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Detalles Bibliográficos
Autores principales: Lenders, Malte, Schmitz, Boris, Brand, Stefan-Martin, Brand, Eva
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Letter to the Editor
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6162957/
https://www.ncbi.nlm.nih.gov/pubmed/30268124
http://dx.doi.org/10.1186/s13023-018-0916-1
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author Lenders, Malte
Schmitz, Boris
Brand, Stefan-Martin
Brand, Eva
author_facet Lenders, Malte
Schmitz, Boris
Brand, Stefan-Martin
Brand, Eva
author_sort Lenders, Malte
collection PubMed
description 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.
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spelling pubmed-61629572018-10-04 Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact? Lenders, Malte Schmitz, Boris Brand, Stefan-Martin Brand, Eva Orphanet J Rare Dis Letter to the Editor 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. BioMed Central 2018-09-29 /pmc/articles/PMC6162957/ /pubmed/30268124 http://dx.doi.org/10.1186/s13023-018-0916-1 Text en © The Author(s). 2018 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.
spellingShingle Letter to the Editor
Lenders, Malte
Schmitz, Boris
Brand, Stefan-Martin
Brand, Eva
Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?
title Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?
title_full Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?
title_fullStr Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?
title_full_unstemmed Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?
title_short Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?
title_sort neutralizing anti-drug antibodies in fabry disease have no obvious clinical impact?
topic Letter to the Editor
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6162957/
https://www.ncbi.nlm.nih.gov/pubmed/30268124
http://dx.doi.org/10.1186/s13023-018-0916-1
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