Urea Cycle Disorders

Also known as: Urea Cycle Disorder / Disorder of the Urea Cycle / Disorders of urea cycle metabolism / Disorder of urea cycle metabolism, unspecified / Urea Cycle Disorders, Inborn / Disorder of the urea cycle metabolism (disorder)

Drug	Drug Name	Drug Description
DB00125	Arginine	An amino acid commonly found as a component of total parenteral nutrition.
DB08909	Glycerol phenylbutyrate	A nitrogen-binding agent used to manage patients with urea cycle disorders who cannot be sufficiently managed amino acid supplementation and/or dietary restrictions.
DB06819	Phenylbutyric acid	An 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.

Drug	Drug Name	Target	Type
DB00125	Arginine	Cationic amino acid transporter 3	target
DB00125	Arginine	Arginine decarboxylase	target
DB00125	Arginine	Argininosuccinate lyase	target
DB00125	Arginine	Nitric oxide synthase, inducible	target
DB00125	Arginine	High affinity cationic amino acid transporter 1	target
DB00125	Arginine	Arginase-2, mitochondrial	target
DB00125	Arginine	Cationic amino acid transporter 4	target
DB00125	Arginine	Argininosuccinate synthase	target
DB00125	Arginine	Nitric oxide synthase, endothelial	target
DB00125	Arginine	Solute carrier family 22 member 5	transporter
DB00125	Arginine	Monocarboxylate transporter 10	transporter
DB00125	Arginine	Solute carrier family 22 member 4	transporter
DB08909	Glycerol phenylbutyrate	Pancreatic triacylglycerol lipase	enzyme
DB08909	Glycerol phenylbutyrate	Cytochrome P450 3A4	enzyme
DB08909	Glycerol phenylbutyrate	Cytochrome P450 2D6	enzyme
DB06819	Phenylbutyric acid	Aromatic-amino-acid aminotransferase	target
DB06819	Phenylbutyric acid	Histone deacetylase	target

Drug	Drug Name	Phase	Status	Count
DB08909	Glycerol phenylbutyrate	1	Completed	1
DB17667	SYNB1020	1	Completed	1
DB00551	Acetohydroxamic acid	1 / 2	Terminated	1
DB17695	Heterologous human adult liver derived progenitor cells	1 / 2	Completed	1
DB00125	Arginine	2	Completed	1
DB06170	Glycerol phenylbutyrate	2	Completed	1
DB08909	Glycerol phenylbutyrate	2	Completed	1
DB17695	Heterologous human adult liver derived progenitor cells	2	Completed	2
DB17695	Heterologous human adult liver derived progenitor cells	2	Withdrawn	1
DB06819	Phenylbutyric acid	2	Completed	2
DB06170	Glycerol phenylbutyrate	3	Completed	2
DB08909	Glycerol phenylbutyrate	3	Completed	1
DB06819	Phenylbutyric acid	3	Completed	1
DB08909	Glycerol phenylbutyrate	4	Completed	3
DB06819	Phenylbutyric acid	4	Completed	1