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Successful hematopoietic stem cell transplantation in Farber disease

Lookup NU author(s): Professor Hermann Josef Vormoor

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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.

Publication metadata

Author(s): Vormoor J, Ehlert K, Groll AH, Koch HG, Frosch M, Roth J

Publication type: Article

Publication status: Published

Journal: The Journal of Pediatrics

Year: 2004

Volume: 144

Issue: 1

Pages: 132-134

Print publication date: 01/01/2004

ISSN (print): 0022-3476

ISSN (electronic): 1097-6833

URL: http://dx.doi.org/10.1016/j.jpeds.2003.09.051

DOI: 10.1016/j.jpeds.2003.09.051

Notes: 0022-3476 (Print) Case Reports Journal Article

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