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J Pediatr. 2004 Jan;144(1):132-4.

Successful hematopoietic stem cell transplantation in Farber disease.

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1
Department of Pediatric Hematology and Oncology and the Department of Pediatrics, University Children's Hospital Münster, Münster, Germany.

Abstract

Farber disease, a lysosomal storage disorder, has a dismal prognosis leading to death with progressive granulomatous inflammation, even in patients without central nervous system involvement (type 2/3). We report the first successful hematopoietic stem cell transplantations in 2 patients with Farber disease type 2/3, resulting in resolution of symptoms.

PMID:
14722533
DOI:
10.1016/j.jpeds.2003.09.051
[Indexed for MEDLINE]
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