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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Argininosuccinic aciduria


Other Names for this Disease

  • Arginino succinase deficiency
  • Argininosuccinate acidemia
  • Argininosuccinate lyase deficiency
  • Argininosuccinic acid lyase deficiency
  • Argininosuccinicaciduria
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Treatment

Management Guidelines

  • GeneReviews provides current, expert-authored, peer-reviewed, full-text articles describing the application of genetic testing to the diagnosis, management, and genetic counseling of patients with specific inherited conditions. Click on the link to view the article on this topic.
  • Orphanet Emergency Guidelines is an article which is expert-authored and peer-reviewed that is intended to guide health care professionals in emergency situations involving this condition.  

Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Argininosuccinic aciduria. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.
  • The U.S. National Institutes of Health, through the National Library of Medicine, developed ClinicalTrials.gov to provide patients, family members, and members of the public with current information on clinical research studies. There is a study titled Study of Treatment and Metabolism in Patients With Urea Cycle Disorders which may be of interest to you. To find this trial, click on the link above.

Medical Products

The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.


Generic Name Glycerol phenylbutyrate
Trade Name
(Manufacturer Name)
Ravicti
(Hyperion Therapeutics, Inc.)
Indication
The FDA has approved this product to be used in this manner.
Use as a nitrogen-binding adjunctive therapy for chronic management of adult and pediatric patients > or = to 2 years of age with urea cycle disorders (UCDs) that cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (eg, essential amino acids, arginine, citrulline, protein-free calorie supplements).
More Information about this product Drug Information Portal

Other Names for this Disease
  • Arginino succinase deficiency
  • Argininosuccinate acidemia
  • Argininosuccinate lyase deficiency
  • Argininosuccinic acid lyase deficiency
  • Argininosuccinicaciduria
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.