Hereditary Tyrosinemia Type 1 Mice under Continuous Nitisinone Treatment Display Remnants of an Uncorrected Liver Disease Phenotype
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DOI: 10.3390/genes14030693
Abstract: Hereditary tyrosinemia type 1 (HT1) is a genetic disorder of the tyrosine degradation pathway (TIMD) with unmet therapeutic needs. HT1 patients are unable to fully break down the amino acid tyrosine due to a deficient… read more here.
Keywords: ntbc therapy; liver disease; hereditary tyrosinemia; tyrosinemia type ... See more keywords