Taste-masked formulation of sodium phenylbutyrate (ACER-001) for the treatment of urea cycle disorders.
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DOI: 10.2139/ssrn.4291835
Abstract: Urea cycle disorders (UCDs) are a group of rare inherited metabolic diseases caused by a deficiency of one of the enzymes or transporters that constitute the urea cycle. Defects in these enzymes lead to acute… read more here.
Keywords: phenylbutyrate; formulation; urea cycle; acer 001 ... See more keywords