Article
Wolman disease successfully treated by bone marrow transplantation.
Bone marrow transplantation - 1 Sept 2000
Krivit W, Peters C, Dusenbery K, Ben-Yoseph Y, Ramsay N K, Wagner J E, Anderson R
Abstract excerpt
Wolman disease is characterized by severe diarrhea and malnutrition leading to death during infancy. Lysosomal acid lipase deficiency is the cause of the symptoms and signs. It is inherited in an autosomal recessive manner. All Wolman disease patients have adrenal gland calcification. Previous therapeutic attempts have failed to provide remission. We report successful long-term bone marrow engraftment in a...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.