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Citrullinemia type I (CTLN1), also known as arginosuccinate synthetase deficiency, is a rare disease caused by a deficiency in argininosuccinate synthetase, an enzyme involved in excreting excess nitrogen from the body. [1] There are mild and severe forms of the disease, which is one of the urea cycle disorders.
An accumulation of ammonia during the first few days of life leads to poor feeding, vomiting, seizures, and the other signs and symptoms of type I citrullinemia. Treatment for this defect includes a low-protein diet and dietary supplementation with arginine and phenylacetate. Arginine allows the urea cycle to complete itself, creating the ...
Citrullinemia is an autosomal recessive urea cycle disorder that causes ammonia and other toxic substances to accumulate in the blood. [1] Two forms of citrullinemia have been described, both having different signs and symptoms, and are caused by mutations in different genes. Citrullinemia belongs to a class of genetic diseases called urea ...
This list of over 500 monoclonal antibodies includes approved and investigational drugs as well as drugs that have been withdrawn from market; consequently, the column Use does not necessarily indicate clinical usage. See the list of FDA-approved therapeutic monoclonal antibodies in the monoclonal antibody therapy page.
Novartis' heart failure drug Entresto cost the Medicare agency $2.9 billion to treat almost 60,000 patients in the same period. ... AbbVie and J&J's leukemia treatment Imbruvica cost the federal ...
Pulmonary surfactant is used as a medication to treat and prevent respiratory distress syndrome in newborn babies. [1] Prevention is generally done in babies born at a gestational age of less than 32 weeks. [1] It is given by the endotracheal tube. [1] Onset of effects is rapid. [2] A number of doses may be needed. [2]
On Thursday, the FDA approved AbbVie Inc’s (NYSE:ABBV) Vyalev (foscarbidopa and foslevodopa) as the first and only subcutaneous 24-hour infusion of levodopa-based therapy for motor fluctuations ...
Citrullinemia type II [1] Inborn errors of organic acid metabolism. Methylmalonic acidemia (Cbl C,D) [1] Malonic acidemia [1] 2-Methyl 3-hydroxy butyric aciduria [1] Isobutyryl-CoA dehydrogenase deficiency [1] 2-Methylbutyryl-CoA dehydrogenase deficiency [1] 3-Methylglutaconyl-CoA hydratase deficiency [1] Glutaric acidemia type II