Fabry disease: recent advances in pathology, diagnosis, treatment and monitoring
<p>Abstract</p> <p>Background</p> <p>In Fabry disease (α-galactosidase A deficiency) accumulation of Globotriaosylceramide (Gb3) leads to progressive organ failure and premature death. The introduction of enzyme replacement therapy (ERT) was the beginning of a new era i...
| Main Author: | |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2009-10-01
|
| Series: | Orphanet Journal of Rare Diseases |
| Online Access: | http://www.ojrd.com/content/4/1/21 |