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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.
Treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC3+) biliary tract cancer as detected by an FDA approved test [2] Zenocutuzumab: Treatment of adults with advanced, unresectable or metastatic non-small cell lung cancer harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior ...
Lucinactant is listed as an Orphan Drug Product by the US Food and Drug Administration for several conditions: [9] [10] 07-30-1996 Treatment of meconium aspiration syndrome in newborn infants; 07-17-1995 Treatment of acute respiratory distress syndrome in adults. 05-23-2006 Prevention of bronchopulmonary dysplasia in premature infants
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]
This list of over 500 monoclonal antibodies includes approved and investigational drugs as well as drugs that have been withdrawn from market; consequently, the column Use does not necessarily indicate clinical usage.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.
A breakthrough therapy designation can be assigned to a drug if "it is a drug which is intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition" and if the preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as ...
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 ...
The center doesn't actually test drugs itself, although it does conduct limited research in the areas of drug quality, safety, and effectiveness standards. As of the end of 2013, the FDA and its predecessors had approved 1,452 drugs, though not all are still available, and some have been withdrawn for safety reasons. [5]