Abstract: A method for determining a personalized full dose of a melphalan compound (e.g., melphalan) in a reduced intensity conditioning regimen (RIC) prior to hematopoietic cell transplantation for a subject based on pharmacokinetic features of the melphalan compound administered to the subject at a test dose.

Abstract: Markers for genomic instability in Fanconi Anemia (FA) and other pathologies for therapeutic and diagnostic uses. In one embodiment, glycosphingolipid metabolism is altered in the FA deficient squamous cell carcinoma (SCC) cells, based on analysis of a metabolomics/lipidomics platform. The data indicated ganglioside metabolism was important in FA patients' susceptibility to SCC progression.

Abstract: Markers for genomic instability in Fanconi Anemia (FA) and other pathologies for therapeutic and diagnostic uses. In one embodiment, glycosphingolipid metabolism is altered in the FA deficient squamous cell carcinoma (SCC) cells, based on analysis of a metabolomics/lipidomics platform. The data indicated ganglioside metabolism was important in FA patients' susceptibility to SCC progression.

Abstract: One aspect of this invention is directed to a pharmacologically acceptable composition including a sulfate of 3 alpha, 7 beta-dihydroxy-5 beta-cholan-24-oic acid (Ursodeoxycholic acid or “UDCA”) and a pharmacologically acceptable carrier. In a preferred composition, the sulfate is UDCA-3-sulfate, UDCA-7-sulfate, UDCA-3,7-disulfate, glyco-UDCA-3-sulfate, glyco-UDCA-7-sulfate, glyco-UDCA-3,7-disulfate, tauro-UDCA-3-sulfate, tauro-UDCA-7-sulfate, tauro-UDCA-3,7-disulfate or a combinations thereof. Another aspect of the invention concerns a method of delivering UDCA to a mammal to inhibit or treat a disorder, which includes administering a sulfate of UDCA to the mammal in an amount sufficient to inhibit or treat the disorder.

Abstract: This invention concerns a method of delivering UDCA to a mammal to inhibit or treat a disorder, which includes administering a sulfate of UDCA to the mammal in an amount sufficient to inhibit or treat the disorder. For example, a UDCA sulfate may be used to advantage in inhibiting or treating an inflammatory condition of the gastrointestinal tract, such as colon cancer, rectum cancer, a neoplasm of the colon, a neoplasm of the rectum, carcinogenesis of the colon, carcinogenesis of the rectum, ulcerative colitis, an adenomatous polyp, familial polyposis and the like. A sulfate of UDCA also may be administered to inhibit or treat an inflammatory disorder of the liver. A UDCA sulfate may be used to improve serum biochemistries of liver disease or liver function, to increase bile flow or to decrease biliary secretion of phospholipid or cholesterol.