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Posted 1 Apr 2010 by Drugs.com
The U.S. Food and Drug Administration today approved Carbaglu (carglumic acid) Tablets to treat a condition that results in too much ammonia in the blood. The condition, N-acetylglutamate synthase or NAGS deficiency, is an extremely rare, genetic disorder that can be present in babies soon after birth. NAGS deficiency and the resulting elevated levels of ammonia (hyperammonemia) can be fatal if it is not detected and treated rapidly. DNA testing can confirm the diagnosis of NAGS. "We are very excited that more drugs are being developed to treat very rare but often devastating genetic disorders" said Janet Woodcock, M.D., director of FDA's Center for Drug Evaluation and Research. "We hope to see continuing progress in this area." The safety and efficacy of Carbaglu was studied in 23 patients with NAGS who received the treatment for times ranging from six months to 21 years. In these ... Read more
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