Ornithine Transcarbamylase Deficiency

Also known as: Ornithine Carbamoyltransferase Deficiency / Ornithine transcarbamoylase deficiency / Ornithine Transcarbamylase Deficiency Disease / Ornithine Transcarbamylase (OTC) Deficiency / Disorders of ornithine metabolism / Ornithine carbamoyltransferase deficiency (disorder) / Ornithine Carbamoyltransferase Deficiency Disease

DrugDrug NameDrug Description
DB06819Phenylbutyric acidAn agent indicated for the adjunctive therapy for the management of chronic urea cycle disorders due to deficiencies in specific enzymes, including the neonatal-onset deficiency and late-onset disease with a history of hyperammonemic encephalopathy.
DrugDrug NamePhaseStatusCount
DB17683ARCT-8101Active Not Recruiting1
DB00551Acetohydroxamic acid1 / 2Not Yet Recruiting1
DB17656MRT52011 / 2Withdrawn1
DB03904Urea1 / 2Not Yet Recruiting1
DB17683ARCT-8102Active Not Recruiting1
DB06775Carglumic acid2Completed1
DB17695Heterologous human adult liver derived progenitor cells2Completed1