Farber disease: clinical presentation, pathogenesis and a new approach to treatment
<p>Abstract</p> <p>Background</p> <p>Farber Disease is an autosomal-recessively inherited, lysosomal storage disorder caused by acid ceramidase deficiency and associated with distinct clinical phenotypes. Children with significant neurological involvement usually die ea...
| Main Authors: | , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2007-06-01
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| Series: | Pediatric Rheumatology Online Journal |
| Online Access: | http://www.ped-rheum.com/content/5/1/15 |