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Case Reports
. 2004 Jan;144(1):132-4.
doi: 10.1016/j.jpeds.2003.09.051.

Successful hematopoietic stem cell transplantation in Farber disease

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Case Reports

Successful hematopoietic stem cell transplantation in Farber disease

Josef Vormoor et al. J Pediatr. 2004 Jan.

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.

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