E Riccio, M Zanfardino, L Ferreri, C Santoro… - European Journal of …, 2020 - nature.com
… replacementtherapy (ERT) with agalsidase alfa or beta, and the oralpharmacological chaperonemigalastat. … on the effects of switching from ERT to migalastat, we performed a single-…
F Perretta, S Jaurretche - Healthcare, 2023 - mdpi.com
… who were previously on enzymereplacementtherapy and switched to migalastat. In this … of switching from enzymereplacementtherapy to migalastat in patients with Fabry disease and “…
… Migalastat, the oralpharmacologicchaperone … than enzyme-replacementtherapy given every 2 weeks. The higher volume of distribution of migalastat than of enzymereplacement 11 …
… Oralmigalastat is the first pharmacologicalchaperone approved for … Fabry disease who have a migalastat-amenable GLA mutation. In the FACETS trial in enzymereplacementtherapy (…
…, Polish Fabry Disease Collaborative Group - Orphanet journal of rare …, 2024 - Springer
… Disease-specific management of FD includes enzymereplacementtherapy with agalsidase α and β or pharmacologicaloralchaperonemigalastat. Migalastat is a low-molecular-mass …
F Weidemann, A Jovanovic, K Herrmann… - International journal of …, 2022 - mdpi.com
… The usual treatment of FD is enzymereplacementtherapy (ERT)… and since a few years ago chaperonetherapyMigalastat [17]. In … Oralchaperonetherapy with Migalastat in patients with …
J Müntze, D Gensler, O Maniuc, D Liu… - … Pharmacology & …, 2019 - Wiley Online Library
… patients who did not receive Fabry-specific therapy before migalastat (therapy-naive group; … from enzymereplacementtherapy to migalastat (previous enzymereplacementtherapy …
D Oder, J Müntze, P Nordbeck - … Diagnosis and Therapy, 2021 - ncbi.nlm.nih.gov
… Thus, the first oralFabry-specific drug in form of the chaperoneMigalastat (Galafold … treatment options In 2020 there are two different Fabry-specific therapy approaches with three drug …
… In summary, migalastat HCl is a candidate pharmacologicalchaperone that provides a … oral small molecule pharmacologicalchaperone, rather than using enzymereplacementtherapy, …