期刊
JOURNAL OF PEDIATRICS
卷 144, 期 1, 页码 132-134出版社
MOSBY-ELSEVIER
DOI: 10.1016/j.jpeds.2003.09.051
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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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