Successful hematopoietic stem cell transplantation in Farber disease.
case_report · Level V
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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.
Medical subject headings
- Galactosylgalactosylglucosylceramidase
- Granuloma
- Hematopoietic Stem Cell Transplantation
- Lysosomal Storage Diseases