Published in

Elsevier, Journal of Biological Chemistry, 34(287), p. 28386-28397, 2012

DOI: 10.1074/jbc.m112.351056

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α-Galactosidase Aggregation Is a Determinant of Pharmacological Chaperone Efficacy on Fabry Disease Mutants

This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Abstract

Background: Deficiency in α-galactosidase activity leads to Fabry disease, for which treatment employing pharmacological chaperones is being developed.