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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Laron syndrome


Other Names for this Disease

  • Growth hormone insensitivity syndrome
  • Growth hormone receptor deficiency
  • Laron dwarfism
  • Laron type pituitary dwarfism I
  • Pituitary dwarfism II
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

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How might Laron syndrome be treated?

Treatment for individuals with Laron syndrome mainly aims at improving growth.[1] The only specific treatment available for this condition is subcutaneous injections of insulin-like growth factor 1 (a growth-promoting hormone), often called IGF-1. IGF-1 stimulates linear growth (height) and improves brain growth as well as the metabolic abnormalities caused by long-term IGF-1 deficiency. It has also been shown to raise blood glucose levels, reduce cholesterol, increase muscle growth, and give other benefits.[2] The growth resulting from IGF-1 treatment is less than what is typically seen with human growth hormone (hGH) treatment in children with growth hormone deficiency. Treatment with IGF-1 reportedly stimulates growth by about 8cm in the first year and 4-5cm in the following years,[3] but more studies on the long-term effects on final height are needed. IGF-1 and GH levels should be closely monitored in individuals undergoing this treatment, because overdosage of IGF-I causes a variety of health problems.[4] Infants may also need frequent feedings in order to avoid hypoglycemia.[1]
Last updated: 11/3/2011

References
  1. J. L├ęger. Laron syndrome. Orphanet. November 2009; http://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=633. Accessed 11/2/2011.
  2. Zvi Laron. Growth Hormone Insensitivity (Laron Syndrome). Reviews in Endocrine & Metabolic Disorders. 2002; 3:347-355.
  3. Laron Z. The essential role of IGF-I: lessons from the long-term study and treatment of children and adults with Laron syndrome. J Clin Endocrinol Metab. December 1999; 84(12):4397-4404.
  4. Laron Z. Insulin-like growth factor-I treatment of children with Laron syndrome (primary growth hormone insensitivity). Pediatr Endocrinol Rev. March 2008; 5(3):766-771.


Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Laron syndrome. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.

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 Mecasermin
Trade Name
(Manufacturer Name)
Increlex®
(Tercica, Inc.)
Indication
The FDA has approved this product to be used in this manner.
Long-term treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to growth hormone.
More Information about this product Drug Information Portal

Generic Name Mecasermin rinfabate
Trade Name
(Manufacturer Name)
Iplex®
(Insmed, Inc.)
Indication
The FDA has approved this product to be used in this manner.
Treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to growth hormone
More Information about this product Drug Information Portal

Other Names for this Disease
  • Growth hormone insensitivity syndrome
  • Growth hormone receptor deficiency
  • Laron dwarfism
  • Laron type pituitary dwarfism I
  • Pituitary dwarfism II
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.