| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10091658 | The Journal of Pediatrics | 2005 | 4 Pages |
Abstract
IPEX (immune-dysregulation, polyendocrinopathy, enteropathy, X-linked) syndrome is an autoimmune disorder with an often lethal outcome in spite of immunosuppressive therapy. We report the successful use of sirolimus in 3 patients with IPEX. The efficacy of sirolimus is probably due to its different mode of action compared to calcineurin-dependent agents.
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Authors
Lutz MD, Troy MD, PhD, Lucia MD, Nelly MD, Hans D. MD, Olivier MD, Frank M. MD, PhD,