TY  - JOUR
AB  - 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.
AU  - Lenders, Malte
AU  - Schmitz, Boris
AU  - Brand, Stefan-Martin
AU  - Brand, Eva
DA  - 2018-09-29
DO  - 10.1186/s13023-018-0916-1
KW  - Enzyme replacement therapy
KW  - Longitudinal
KW  - Prospective
LA  - eng
N1  - Orphanet Journal of Rare Diseases 13 (2018 ) 171, 1-2
N1  - Finanziert durch den Open-Access-Publikationsfonds 2018 der Deutschen Forschungsgemeinschaft (DFG) und der Westfälischen Wilhelms-Universität Münster (WWU Münster).
PY  - 2018-09-29
TI  - Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?
UR  - https://nbn-resolving.org/urn:nbn:de:hbz:6-84129538247
Y2  - 2024-11-22T02:16:25
ER  -