Ten-year-long enzyme replacement therapy shows a poor effect in alleviating giant leg ulcers in a male with Fabry disease

Ten-year-long enzyme replacement therapy shows a poor effect in alleviating giant leg ulcers in a male with Fabry disease

Fabry disease is an X-linked lysosomal storage disorder caused by a deficiency of α-galactosidase A (α-gal A), leading to the progressive accumulation of glycosphingolipids. Classical hemizygous males usually present symptoms, including pain and paresthesia in the extremities, angiokeratoma, hypo- o...

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Bibliographic Details
Main Authors: Jun Okada, Mohammad Arif Hossain, Chen Wu, Takashi Miyajima, Hiroko Yanagisawa, Keiko Akiyama, Yoshikatsu Eto
Format: Article
Language:English
Published: Elsevier 2018-03-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
α-Galactosidase A
Enzyme replacement therapy
Leg ulcer
Zebra bodies
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426917301349

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