From Our 2010 Archives
Carbaglu Approved for Rare Genetic Disorder
Latest Medications News
THURSDAY, March 18 (HealthDay News) -- The drug Carbaglu (carglumic acid) has been approved by the U.S. Food and Drug Administration to treat a rare genetic disorder called N-acetylglutamate synthase (NAGS deficiency), which results in elevated blood levels of ammonia, the agency said Thursday.
The drug's safety and effectiveness were evaluated in 23 people with NAGS. Carbaglu reduced blood ammonia within a day and lowered levels to a normal range within three days, the FDA said.
The drug should only be administered by a doctor with experience in treating metabolic problems, the agency said. The medication is manufactured by Orphan Europe.
-- Scott Roberts
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