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FABRY DISEASE: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat. What Do We Know Today?

2022-12-01

Abstract excerpt

Fabry disease is a lysosomal storage disorder caused by the deficiency of the α-galactosidase-A enzyme. Cardiac, renal, and neurological involvement significantly reduces life expectancy. Until a few years ago, treatment options for Fabry disease were limited to enzyme replacement therapy with agalsidase alfa or beta administered by intravenous infusion every 2 weeks. Migalastat (Galafold®) is an oral ph...

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Identifiers and source

Literature Corpus work
b3743a1d-f515-51e1-b126-dfc5bc45e0f1
DOI
10.20944/preprints202212.0022.v1
Open publication

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FABRY DISEASE: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat. What Do We Know Today?DOI 10.20944/preprints202212.0022.v1
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