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HEREDITARY orotic aciduria is a disorder of pyrimidine metabolism manifested by megaloblastic anemia, leukopenia, retarded growth and the urinary excretion of large quantities of orotic acid.1 , 2 ...
Xuriden (uridine triacetate; Wellstat Therapeutics) has been approved for the treatment of hereditary orotic aciduria, the first approved treatment for this rare metabolic disorder.
Cecilia had hereditary orotic aciduria. "They were very honest, like, we've never had a patient with this," Rebekah Shasky said.
Hereditary orotic aciduria is a rare metabolic disorder, which has been reported in approximately 20 patients worldwide. 9 Oct 2015 Trending Stories Latest Interviews Top Health Articles Huw Rees ...
US regulators have green-lighted Xuriden, the first therapy for patients with the ultra rare metabolic disorder hereditary orotic aciduria.
Aim: In this study; orotic acid levels in the milk of smoking and non-smoking mothers were investigated by high-performance liquid chromatography (HPLC).
Wellstat Therapeutics Corp. gained a rare pediatric disease priority review voucher (PRV) from the FDA with the agency's approval of Xuriden (uridine triacetate), a pyrimidine analogue for uridine ...
Proof of concept has been achieved for a therapy for argininosuccinic aciduria consisting of human argininosuccinic lyase (ASL) mRNA encapsulated in lipid nanoparticles (ASL-LNPs).
Neven et al. (June 28 issue) 1 discuss the use of bone marrow transplantation in the treatment of severe mevalonic aciduria. We provide additional evidence for the effective cure of mevalonic ...
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