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Carbamoyl-phosphate synthase [ammonia], mitochondrial

2

Histamine H1 receptor

315

Dopamine D2 receptor

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DNA

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Bacterial penicillin-binding protein

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2018

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Acute hyperammonemia

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Orphan Europe

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OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS

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Amino acids and derivatives

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Showing 1 - 2 of 2 results

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CARGLUMIC ACID

5L0HB4V1EW

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Status:

US Approved Rx (2021)

First approved in 2010

Class (Stereo):

CHEMICAL (ABSOLUTE)

Targets:

Carbamoyl-phosphate synthase [ammonia], mitochondrial

Conditions:

Acute hyperammonemia

Chronic hyperammonemia

Carglumic acid is a Carbamoyl Phosphate Synthetase 1 (CPS 1) allosteric modulator. CPS1 is found in the mitochondria and is the first enzyme of the urea cycle, which converts ammonia into urea. Carglumic acid acts as a replacement for NAG in NAGS def...

iciency patients by activating CPS1 but it does not help to regulate the urea cycle. Carglumic acid under the trade name Carbaglu indicated as adjunctive therapy for the treatment of acute hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS). In addition, as maintenance therapy for the treatment of chronic hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS). This rare genetic disorder results in elevated blood levels of ammonia, which can eventually cross the blood–brain barrier and cause neurologic problems, cerebral edema, coma, and death.

ACETYL GLUTAMIC ACID

MA61H539YZ

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Status:

Possibly Marketed Outside US

First approved in 2018

Class (Stereo):

CHEMICAL (ABSOLUTE)

Targets:

Carbamoyl-phosphate synthase [ammonia], mitochondrial

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