Medical uses<unk>  is taken orally or by nasogastric intubation as a tablet or powder, and tastes very salty and bitter. It treats urea cycle disorders, genetic diseases in which nitrogen waste builds up in the blood plasma as ammonia glutamine (a state called hyperammonemia) due to deficiences in the enzymes carbamoyl phosphate synthetase I, ornithine transcarbamylase, or argininosuccinic acid synthetase. Uncontrolled, this causes mental retardation and early death.<unk>  metabolites allows the kidneys to excrete excess nitrogen in place of urea, and coupled with dialysis, amino acid supplements and a protein-restricted diet, children born with urea cycle disorders can usually survive beyond 12 months. Patients may need treatment for all their life. The treatment was introduced by researchers in the 1990s, and approved by the U.S. Food and Drugs Administration (FDA) in April 1996. Adverse effects Nearly of women may experience an adverse effect of amenorrhea or menstrual dysfunction. Appetite loss is seen is 4% of patients. Body odor due to metabolization of<unk>  affects 3% of patients, and 3% experience unpleasant tastes. Gastrointestinal symptoms and mostly mild indications of neurotoxicity are also seen in less than 2% of patients, among several other reported adverse effects. Administration during pregnancy is not recommended because<unk>  treatment could mimic maternal phenylketonuria due to the production of phenylalanine, potentially causing fetal brain damage. Research Urea cycle disorders<unk>  administration was discovered to lead to an alternative nitrogen disposal pathway by Dr. Saul Brusilow, Mark Batshaw and colleagues at the Johns Hopkins School of Medicine in the early 1980s, due to some serendipitous discoveries. They had studied ketoacid therapy for another inborn error of metabolism, citrullinemia, in the late 1970s and they noticed that arginine treatment led to an increase of nitrogen in the urine and a drop in ammonia in the blood. The researchers spoke to Norman Radin about this finding, and he remembered a 1914 article on using<unk>  benzoate to reduce urea excretion. Another 1919 article had used<unk>  phenylacetate, and so the researchers treated 5 patients with hyperammonemia with benzoate and phenylacetate and published a report in Science. In 1982 and 1984, the researchers published on using benzoate and arginine for urea cycle disorders in the NEJM. Use of<unk>  was introduced in the early 1990s, as it lacks the odor of phenylacetate. Chemical chaperone In cystic fibrosis, a point mutation in the Cystic Fibrosis Transmembrane Conductance Regulator protein, ΔF508-CFTR, causes it to be unstable and misfold, hence trapped in the endoplasmic reticulum and unable to reach the cell membrane. This lack of CFTR in the cell membrane leads to disrupted chloride transport and the symptoms of cystic fibrosis.<unk>  can act as a chemical chaperone, stabilising the mutant CFTR in the endoplasmic reticulum and allowing it to reach the cell surface. Histone deacetylase inhibitor Deriving from its activity as a histone deacetylase inhibitor,<unk>  is under investigation for use as a potential differentiation-inducing agent in malignant glioma and acute myeloid leukaemia, and also for the treatment of some sickle-cell disorders as an alternative to hydroxycarbamide due it inducing expression of fetal hemoglobin to replace missing adult hemoglobin. While small-scale investigation is proceeding, there is to date no published data to support the use of the compound in the clinical treatment of cancer, and it remains under limited investigation.<unk>  is also being studied as a therapeutic option for the treatment of Huntington's disease. Other<unk>  has been associated with longer lifespans in Drosophila. University of Colorado researchers Dr. Curt Freed and Wenbo Zhou demonstrated that<unk>  stops the progression of Parkinson's disease in mice by turning on a gene called DJ-1 that can protect dopaminergic neurons in the midbrain from dying. they plan on testing<unk>  for the treatment of Parkinson's disease in humans.
Sodium phenylbutyrate
Sodium fluoroacetate
Doisynolic acid
Kaurenoic acid
Glucosamine
Tuaminoheptane
Geritol
Testosterone nicotinate