Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT …

DA Hughes, K Nicholls, SP Shankar… - Journal of medical …, 2017 - jmg.bmj.com
Migalastat, an oral pharmacological chaperone being developed as an alternative to
intravenous enzyme replacement therapy … of migalastat on renal function in patients with Fabry

Switch from enzyme replacement therapy to oral chaperone migalastat for treating fabry disease: real-life data

E Riccio, M Zanfardino, L Ferreri, C Santoro… - European Journal of …, 2020 - nature.com
replacement therapy (ERT) with agalsidase alfa or beta, and the oral pharmacological
chaperone migalastat. … on the effects of switching from ERT to migalastat, we performed a single-…

Fabry disease: switch from enzyme replacement therapy to oral chaperone migalastat: what do we know today?

F Perretta, S Jaurretche - Healthcare, 2023 - mdpi.com
… who were previously on enzyme replacement therapy and switched to migalastat. In this …
of switching from enzyme replacement therapy to migalastat in patients with Fabry disease and “…

[HTML][HTML] Treatment of Fabry's disease with the pharmacologic chaperone migalastat

DP Germain, DA Hughes, K Nicholls… - New england journal …, 2016 - Mass Medical Soc
Migalastat, the oral pharmacologic chaperone … than enzyme-replacement therapy given
every 2 weeks. The higher volume of distribution of migalastat than of enzyme replacement 11 …

Migalastat: a review in Fabry disease

EH McCafferty, LJ Scott - Drugs, 2019 - Springer
Oral migalastat is the first pharmacological chaperone approved for … Fabry disease who
have a migalastat-amenable GLA mutation. In the FACETS trial in enzyme replacement therapy (…

A review and recommendations for oral chaperone therapy in adult patients with Fabry disease

…, Polish Fabry Disease Collaborative Group - Orphanet journal of rare …, 2024 - Springer
… Disease-specific management of FD includes enzyme replacement therapy with
agalsidase α and β or pharmacological oral chaperone migalastat. Migalastat is a low-molecular-mass …

Chaperone therapy in Fabry disease

F Weidemann, A Jovanovic, K Herrmann… - International journal of …, 2022 - mdpi.com
… The usual treatment of FD is enzyme replacement therapy (ERT)… and since a few years ago
chaperone therapy Migalastat [17]. In … Oral chaperone therapy with Migalastat in patients with …

Oral chaperone therapy migalastat for treating Fabry disease: enzymatic response and serum biomarker changes after 1 year

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 enzyme replacement therapy to migalastat (previous enzyme replacement therapy

[HTML][HTML] Contemporary therapeutics and new drug developments for treatment of Fabry disease: a narrative review

D Oder, J Müntze, P Nordbeck - … Diagnosis and Therapy, 2021 - ncbi.nlm.nih.gov
… Thus, the first oral Fabry-specific drug in form of the chaperone Migalastat (Galafold … treatment
options In 2020 there are two different Fabry-specific therapy approaches with three drug

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from …

DP Germain, R Giugliani, DA Hughes, A Mehta… - Orphanet journal of rare …, 2012 - Springer
… In summary, migalastat HCl is a candidate pharmacological chaperone that provides a … oral
small molecule pharmacological chaperone, rather than using enzyme replacement therapy, …