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...
| Authors: | |
|---|---|
| Division/Institute: | FB 13: Biologie |
| Document types: | Article |
| Media types: | Text |
| Publication date: | 2018 |
| Date of publication on miami: | 25.07.2019 |
| Modification date: | 02.05.2022 |
| Edition statement: | [Electronic ed.] |
| Source: | Orphanet Journal of Rare Diseases 13 (2018 ) 171, 1-2 |
| Subjects: | Enzyme replacement therapy; Longitudinal; Prospective |
| DDC Subject: | 610: Medizin und Gesundheit |
| License: | CC BY 4.0 |
| Language: | English |
| Funding: | Finanziert durch den Open-Access-Publikationsfonds 2018 der Deutschen Forschungsgemeinschaft (DFG) und der Westfälischen Wilhelms-Universität Münster (WWU Münster). |
| Format: | PDF document |
| URN: | urn:nbn:de:hbz:6-84129538247 |
| Other Identifiers: | DOI: 10.1186/s13023-018-0916-1 |
| Permalink: | https://nbn-resolving.de/urn:nbn:de:hbz:6-84129538247 |
| Digital documents: | 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.