October 4, 2015
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"Researchers from the National Institutes of Health have entered into an agreement with biotechnology company Vtesse, Inc., of Gaithersburg, Maryland, to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal storage disor"...




One patient treated with 650 mg/kg/day of carglumic acid developed symptoms characterized as a monosodium glutamate intoxication-like syndrome: tachycardia, profuse sweating, increased bronchial secretion, increased body temperature and restlessness. These symptoms resolved upon reduction of dose.

Repeated oral dosing of carglumic acid at 2000 mg/kg/day was lethal to most neonatal rats within 2-3 days of treatment. In adult rats, a single oral administration of carglumic acid was not lethal at doses up to 2800 mg/kg (1.8 times the maximum recommended starting dose based on a body surface area comparison to adult humans).



Last reviewed on RxList: 8/16/2013
This monograph has been modified to include the generic and brand name in many instances.


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