Various Alimentary Tract and Metabolism Products
- Various Alimentary Tract and Metabolism Products
- Accession Number
- ATC Classification
Drug Drug Description Lipoic acid For nutritional supplementation, also for treating dietary shortage or imbalance. Nitisinone A hydroxyphenylpyruvate dioxygenase inhibitor used as an adjunct to dietary restrictions for the treatment of hereditary tyrosinemia type 1 (HT-1), which causes intolerance to tyrosine containing foods. Sapropterin A cofactor used as an adjunct to phenylalanine restriction in the treatment of phenylketonuria (PKU). Miglustat A glucosylceramide synthase inhibitor used for the management of mild to moderate type I Gaucher disease for patients who are not candidates for whole enzyme replacement. Benzoic acid An antimicrobial food additive. 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. Teduglutide A glucagon-like peptide-2 (GLP-2) analog used to treat patients with Short Bowel Syndrome (SBS) who require parenteral nutritional support. 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. Eliglustat A glucosylceramide synthase used to treat type 1 Gaucher disease in patients who are CYP2D6 extensive, intermediate, or poor metabolizers. Anethole trithione A medication used to treat dry mouth associated with medication or radiotherapy of the head and neck. Zinc acetate A medication used to treat zinc deficiency. Migalastat An alpha-galactosidase A chaperone used for the treatment of Fabry disease in patients with an amenable galactosidase alpha gene (GLA) variant. Triethylenetetramine A copper chelating agent used for the management of Wilson's disease in cases where penicillamine therapy is clinically inappropriate. Uridine triacetate A pyrimidine analog used for the reversal of overdose, regardless of the presence of symptoms, and early-onset, severe or life-threatening CNS toxicity or cardiotoxicity of fluorouracil or capecitabine. Telotristat A tryptophan hydroxylase inhibitor. Telotristat ethyl (brand name Xermelo) is a prodrug of telotristat. Lonafarnib A potent farnesyl transferase inhibitor used to reduce mortality associated with Hutchinson-Gilford progeria syndrome (HGPS) and other progeroid laminopathies. Phenylacetic acid An agent used as an adjunct to treat acute hyperammonemia and associated encephalopathy in adult and pediatric patients with deficiencies in enzymes of the urea cycle. Triheptanoin Triheptanoin is a medium chain triglyceride indicated to provide calories and fatty acids to treat long chain fatty acid oxidation disorders. Givosiran A 5-aminolevulinic acid synthase-directed small interfering RNA (siRNA) used in the prophylaxis of acute hepatic porphyria. Lumasiran Lumasiran is an interfering RNA that silences hydroxyacid oxidase 1 for the treatment of primary hypoxaluria type 1. Fosdenopterin An exogenous form of cyclic pyranopterin monophosphate (cPMP) used as a replacement substrate in patients with molybdenum cofactor deficiency (MoCD) type A. Elivaldogene autotemcel A gene replacement therapy for treating early cerebral adrenoleukodystrophy comprising autologous hematopoietic stem cells transduced with a viral vector encoding ABCD1 complementary DNA for human adrenoleukodystrophy protein.
- Drugs & Drug Targets