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Successful hematopoietic stem cell transplantation in Farber disease
Josef Vormoor1, Karoline Ehlert, Andreas H Groll
1Department of Pediatric Hematology and Oncology and the Department of Pediatrics, University Children's Hospital Münster, Münster, Germany.
The Journal of Pediatrics
|January 15, 2004
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.