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The remarkable journey of one female individual with ornithine transcarbamylase deficiency diagnosed post-mortem.

RaeLynn ForsythRyan H PeretzAngela DempseyJacquelyn BrittonLisa KratzAda HamoshHilary VernonMark L BatshawDavid Valle
Published in: JIMD reports (2023)
Urea cycle disorders (UCDs) comprise a group of inborn errors of metabolism with impaired ammonia clearance and an incidence of ~1:35 000 individuals. First described in the 1970s, the diagnosis and management of these disorders has evolved dramatically. We report on a 59-year-old woman with a UCD who contributed to advances in the understanding and treatment of this group of disorders. This individual was diagnosed with carbamoyl phosphate synthetase 1 deficiency based on a biochemical assay under a research context predating genetic sequencing, treated longitudinally as having this metabolic disorder, and was among the first participants to trial UCD pharmaceutical therapies. She ultimately succumbed to a SARS-CoV-2 infection while maintaining unexpectedly normal ammonium levels. Postmortem genetic testing revealed ornithine transcarbamylase deficiency. This individual's contributions to the field of UCDs is discussed herein.
Keyphrases
  • replacement therapy
  • single cell
  • clinical trial
  • risk factors
  • randomized controlled trial
  • high throughput
  • genome wide
  • copy number
  • study protocol
  • dna methylation
  • smoking cessation
  • phase iii
  • adverse drug