Fabry Disease Therapy: State-of-the-Art and Current Challenges
Fabry disease (FD) is a lysosomal storage disorder caused by mutations of the <i>GLA</i> gene that lead to a deficiency of the enzymatic activity of α-galactosidase A. Available therapies for FD include enzyme replacement therapy (ERT) (agalsidase alfa and agalsidase beta) and the chaper...
Main Authors: | , , , , |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2021-12-01
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Series: | International Journal of Molecular Sciences |
Subjects: | |
Online Access: | https://www.mdpi.com/1422-0067/22/1/206 |