Enzyme Replacement Therapy for Fabry Disease
Fabry disease is a rare X-linked disease caused by the deficiency of α-galactosidase that leads to the accumulation of abnormal glycolipid. Untreated patients develop potentially lethal complications by age 30 to 50 years. Enzyme replacement therapy is the current standard of therapy for Fabry disea...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
SciELO
2016-11-01
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| Series: | Journal of Inborn Errors of Metabolism and Screening |
| Online Access: | https://doi.org/10.1177/2326409816679428 |