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1 results about "Dihydropyrimidinuria" patented technology
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A number sign (#) is used with this entry because of evidence that dihydropyrimidinase deficiency is caused by homozygous or compound heterozygous mutation in the DPYS gene ... DPYS deficiency is an autosomal recessive disease characterized by the presence of dihydropyrimidinuria. The clinical phenotype is highly variable, ranging from early ...
Dihydropyrimidine-2,4(1H,3H)-dione-containing polycyclic derivative, pharmaceutical composition thereof, method of preparation thereof, and uses thereof
This disclosure relates to dihydropyrimidine-2,4(1H,3H)-dione-containing polycyclic compounds or pharmaceutically acceptable salts thereof and compositions thereof that effectively reduce or regulate the expression level of broad-space zinc finger motif (WIZ) proteins and / or induce the expression of fetal hemoglobin (HbF), as well as methods for preparing them and their use in the treatment of subjects requiring them.
Owner:LINKCURE THERAPEUTICS