Key considerations for measuring α-galactosidase A activity after long-term migalastat therapy-Avoiding in vitro inhibition effects - PubMed
5 hours ago
- #Fabry disease
- #migalastat
- #α-galactosidase A
- Assessment of α-galactosidase A (α-GAL) activity is a reliable method for evaluating migalastat efficacy in Fabry disease patients.
- Approximately 30% of Japanese Fabry patients respond to migalastat, showing measurable enzyme activity post-treatment.
- Dried blood spots (DBSs) are used for periodic α-GAL activity measurement, but residual migalastat can inhibit the assay.
- Using Amicon®Ultra filters to remove migalastat from DBS samples enables accurate α-GAL activity assessment.
- The study highlights the importance of avoiding in vitro inhibition effects for accurate long-term therapeutic monitoring.