Laron Syndrome (Syndrome, Laron)

An autosomal recessive disorder characterized by short stature, defective GROWTH HORMONE RECEPTOR, and failure to generate INSULIN-LIKE GROWTH FACTOR I by GROWTH HORMONE. Laron syndrome is not a form of primary pituitary dwarfism (GROWTH HORMONE DEFICIENCY DWARFISM) but the result of mutation of the human GHR gene on chromosome 5.

Also Known As:

Networked: 235 relevant articles (2 outcomes, 26 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1.	Laron Syndrome (Syndrome, Laron)
2.	Multiple Sclerosis
3.	Amyotrophic Lateral Sclerosis (Lou Gehrig's Disease)
4.	Alzheimer Disease (Alzheimer's Disease)
5.	Obesity

Experts

1.	Laron, Zvi: 32 articles (12/2015 - 01/2002)
2.	Laron, Z: 15 articles (08/2011 - 01/2000)
3.	Shevah, Orit: 4 articles (05/2014 - 03/2004)
4.	Jorge, Alexander A L: 4 articles (05/2014 - 01/2004)
5.	Lilos, Pearl: 4 articles (01/2014 - 04/2003)
6.	Kornreich, L: 4 articles (03/2009 - 04/2002)
7.	Kemp, Stephen F: 4 articles (03/2007 - 04/2005)
8.	Shevah, O: 4 articles (02/2007 - 07/2002)
9.	Arnhold, Ivo J P: 3 articles (05/2014 - 01/2004)
10.	Rosenfeld, Ron G: 3 articles (05/2014 - 06/2007)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Laron Syndrome:

1.	Insulin-Like Growth Factor I (IGF-1)IBA 12/01/1999 - "This study reveals that IGF-I treatment at a dose of 80 microg/kg twice daily is effective in patients with growth hormone insensitivity syndrome. " 06/01/2012 - "Recombinant human IGF-I is an effective treatment of severe primary IGF deficiency, which is typical of patients with GH receptor defects (Laron syndrome). " 02/01/2012 - "The study group consisted of 11 patients with Laron syndrome: 5 untreated adults, 5 children and young adults treated with replacement IGF-I starting at bone age <2 years, and 1 adolescent who started replacement therapy at bone age 4.6 years. " 02/01/2012 - "The aim of this prospective clinical study was to test auditory function in patients with Laron syndrome, either untreated or treated with insulin-like growth factor I (IGF-I). " 03/01/2009 - "Nine untreated adult subjects with Laron Syndrome (6 female subjects, 3 male subjects) aged 28-53 years and 4 girls with Laron Syndrome treated by insulin-like growth factor-I (IGF-I) 120-150 μg/kg/d were included in the study. "
2.	Growth Hormone (Somatotropin)IBA 01/01/2014 - "Six patients (5 with Laron syndrome and 1 with type 1A growth hormone deficiency) were followed for a mean (±SD) of 8.2 ± 1.8 years. " 05/01/2012 - "Growth hormone insensitivity syndrome (GHIS) is characterized by extreme short stature and resistance to the actions of growth hormone (GH). " 01/01/2010 - "Laron syndrome (LS), known as growth hormone (GH) receptor deficiency, is a rare form of inherited GH resistance. " 09/01/2009 - "The type specimen (LB1) of Homo floresiensis has been hypothesized to be a pathological human afflicted with Laron Syndrome (LS), a type of primary growth hormone insensitivity (Hershkovitz et al.: Am J Phys Anthropol 134 [2007] 198-208). " 04/01/2008 - "Growth hormone (GH) insensitivity syndrome (Laron syndrome) is known to be caused by genetic disorders of the GH-IGF-1 axis. "
3.	Somatotropin Receptors (Growth Hormone Receptor)IBA 05/01/2014 - "Laron syndrome (LS) is a genetic disorder caused by mutations in the growth hormone receptor (GHR) gene. " 01/01/2011 - "The genetic analysis showed that the patient was homozygous for the R217X mutation in the growth hormone receptor gene, which is characteristic of Laron syndrome. " 04/01/2010 - "Novel growth hormone receptor gene mutation in a patient with Laron syndrome." 08/01/2007 - "Laron syndrome is an autosomal recessive disorder caused by defects of growth hormone receptor (GHR) gene. " 08/01/2007 - "Clinical features and growth hormone receptor gene mutations of patients with Laron syndrome from a Chinese family."
4.	Insulin (Novolin)FDA Link 12/01/2015 - "To describe the characteristics of untreated and recombinant insulin-like growth factor 1 (IGF-1)- treated patients with the Laron syndrome (LS) as seen in our clinic over a period of over 50 years. " 01/01/2014 - "Further observations on the effects of long-term treatment with recombinant human insulin-like growth factor 1 in growth hormone insensitivity syndrome." 01/01/2011 - "Children with growth hormone insensitivity syndrome (GHIS) who receive insulin-like growth factor 1 (IGF-1) treatment and enter puberty with inadequate height gain are unlikely to reach adult height within the normal range. " 01/01/2011 - "On the basis of these results, the diagnosis of primary growth hormone insensitivity syndrome was made, and recombinant insulin-like growth factor 1 therapy was initiated. " 01/01/2011 - "Over-stimulation of insulin/IGF-1 signaling by western diet may promote diseases of civilization: lessons learnt from laron syndrome."
5.	Fibrinogen (Factor I)FDA Link 07/01/2009 - "This study evaluates the effects of 8 years of insulinlike growth factor-I therapy on tooth development in patients with growth hormone insensitivity syndrome. " 07/01/2006 - "Insulinlike growth factor I affects ocular development: a study of untreated and treated patients with Laron syndrome." 07/01/2009 - "Treatment of growth hormone insensitivity syndrome with insulinlike growth factor-I appears to lead to an increase in dental maturation, particularly in younger patients. " 01/01/2008 - "Laron syndrome is an autosomal recessive disorder of GH insensitivity that results in decreased insulinlike growth factor-I (IGF-I) synthesis and growth failure. " 07/01/2006 - "To evaluate the ocular dimensions in patients with primary growth hormone receptor insensitivity (Laron syndrome [LS]) and to study the effect of supplemental insulinlike growth factor I (IGF-I) on ocular growth. "
6.	somatotropin-binding protein (growth hormone-binding protein)IBA 01/01/1998 - "We have previously described two families (H and M) with GH binding protein-positive Laron Syndrome (LS), proposed to have one or more post GHR signaling defects. " 12/01/1997 - "A minority of patients with Laron syndrome have normal serum GH binding protein (GHBP), indicating that the defect is elsewhere than in the extracellular domain of the GH receptor. " 05/01/1997 - "Girls with Laron syndrome having positive growth hormone binding protein (GHBP) are less retarded in height than those lacking GHBP." 08/01/1995 - "Patients with idiopathic short stature have decreased serum levels of the GH receptor-related GH-binding protein (GHBP), and low GHBP levels are associated with complete GH insensitivity (Laron) syndrome. " 06/01/1994 - "In Laron syndrome, growth retardation is caused by GH resistance due to GH receptor (GH-R) defects, which are associated in most cases with absent or low serum concentrations of the GH-R-related GH-binding protein (GHBP). "
7.	HormonesIBA 04/01/2011 - "Only patients with an ascertained diagnosis of either Laron syndrome (LS), congenital IGHD, congenital multiple pituitary hormone deficiency (cMPHD) including GH or GHRHR defect were included in this study. " 01/01/2002 - "Evidence is presented that isolated GH deficiency (IGHD), multiple pituitary hormone deficiencies (MPHD) including GH, as well as primary IGE1 deficiency (GH resistance, Laron syndrome) present signs of early ageing such as thin and wrinkled skin, obesity, hyperglycemia and osteoporosis. " 08/01/1998 - "In two patients with growth hormone (GH) insensitivity syndrome (Laron syndrome), in whom the GH receptor is able to bind the hormone, the D152H mutation was identified, and lack of dimerization was proposed to explain GH resistance in these patients. " 01/01/1997 - "The difference in effectiveness between hGH and IGF-I in restoring muscle force may be due to either the more marked muscle underdevelopment in Laron syndrome patients than in patients with IGHD or a difference in action potential between the two hormones." 03/01/1993 - "Lack of hormone binding in COS-7 cells expressing a mutated growth hormone receptor found in Laron dwarfism."
8.	Carrier Proteins (Binding Protein)IBA 12/01/1997 - "We have evaluated the effect of long-term IGF-I treatment on serum IGF-binding protein (IGFBP)-3 and the acid-labile subunit (ALS) in three sibling with Laron syndrome caused by a GH post-receptor defect and with normal GHBP. " 11/25/1997 - "A mammalian model for Laron syndrome produced by targeted disruption of the mouse growth hormone receptor/binding protein gene (the Laron mouse)." 06/01/1994 - "We have selected GHRD (Laron syndrome) as the prototypic disorder of GHI, in part because such dramatic and rapid progress has been made in this clinical condition over the last 6 yr. These advances represent the fortunate convergence of: 1) the cloning of the GHR gene and the identification of deletions and mutations of this gene in GHRD; 2) the development of assay methods for measurement of the GHBP, IGF peptides, and binding proteins; 3) the discovery of a larger number of affected individuals than had been previously suspected, including the recognition and description of a large genetically homogeneous population of GHRD patients in Ecuador; and 4) the production of recombinant IGF-I for therapeutic trials in GHRD. " 03/01/2007 - "In addition, the serum half-life of unbound rhIGF-I is shorter when administered to patients with growth hormone insensitivity syndrome, who have low serum concentrations of its binding proteins IGFBP-3 and acid-labile subunit, than when administered to healthy volunteers or to patients with an IGF-I gene deletion who have normal levels of IGFBP-3. " 04/01/2003 - "SomatoKine, a recombinant fusion of insulin-like growth factor-1 and its binding protein, BP-3, is being developed by Insmed (formerly Celtrix Pharmaceuticals) for the potential treatment of growth hormone insensitivity syndrome (GHIS), and as a potential injectable insulin sensitizer for the management of type 1 and type 2 diabetes in patients who are less sensitive to insulin therapy. "
9.	PeptidesIBA 06/01/1994 - "We have selected GHRD (Laron syndrome) as the prototypic disorder of GHI, in part because such dramatic and rapid progress has been made in this clinical condition over the last 6 yr. These advances represent the fortunate convergence of: 1) the cloning of the GHR gene and the identification of deletions and mutations of this gene in GHRD; 2) the development of assay methods for measurement of the GHBP, IGF peptides, and binding proteins; 3) the discovery of a larger number of affected individuals than had been previously suspected, including the recognition and description of a large genetically homogeneous population of GHRD patients in Ecuador; and 4) the production of recombinant IGF-I for therapeutic trials in GHRD. " 01/01/2005 - "In classical GH insensitivity syndrome (GHIS), known as Laron syndrome, due to GH receptor (GHR) deficiency, serum IGF-I, IGFBP-3 and ALS are severely reduced with inability to produce these peptides during an IGF-I generation test. "
10.	Dihydrotestosterone (Androstanolone)IBA 01/01/2004 - "The study describes for the first time, a homozygous mutation in the WSXWS-like motif of the human GH receptor (GHR) in a patient with Laron syndrome and describe laboratory data during treatment with GnRHa to suppress puberty and dihydrotestosterone (DHT). " 01/01/2004 - "The first homozygous mutation (S226I) in the highly-conserved WSXWS-like motif of the GH receptor causing Laron syndrome: supression of GH secretion by GnRH analogue therapy not restored by dihydrotestosterone administration."

Therapies and Procedures

1.	Subcutaneous Injections 10/01/1992 - "We studied the effects of 9 months of treatment with twice-daily subcutaneous injections of insulin-like growth factor I (IGF-I), 120 micrograms/kg per dose, in a 9.7-year-old child with growth hormone insensitivity syndrome, in whom short-term studies had suggested that IGF-I might promote linear growth. " 07/01/1995 - "Nine prepubertal children with Laron syndrome (6 males, 3 females) aged 0.5 to 14.6 years were treated by daily subcutaneous injections of IGF-I in doses of 150-200 micrograms/kg. All patients completed at least one year of treatment; six completed two years and five three years. " 12/01/1994 - "Eight children with Laron syndrome (5 males, 3 females) aged 3-14.5 years received daily subcutaneous injections of 150 micrograms/kg recombinant insulin-like growth factor-I (IGF-I) for 5 months. "
2.	Transplants (Transplant) 01/10/2013 - "In this study, we have demonstrated that a single intraperitoneal graft of microencapsulated pig Sertoli cells, producing pig insulin-like growth factor-1, successfully promoted significant proportional growth in the Laron mouse, a unique animal model of the human Laron Syndrome. "
3.	Injections 03/01/2013 - "Comparison of the growth velocity of 1 vs. 2 IGF-I injections per day in patients with Laron Syndrome (primary GH insensitivity) revealed similar effects." 02/01/1999 - "Eight children with growth hormone insensitivity syndrome (GHIS) have been treated with injections of recombinant human insulin-like growth factor I (rhIGF-I) for more than 5 years each. " 04/01/2003 - "SomatoKine, a recombinant fusion of insulin-like growth factor-1 and its binding protein, BP-3, is being developed by Insmed (formerly Celtrix Pharmaceuticals) for the potential treatment of growth hormone insensitivity syndrome (GHIS), and as a potential injectable insulin sensitizer for the management of type 1 and type 2 diabetes in patients who are less sensitive to insulin therapy. "
4.	Tissue Therapy (Cell Therapy) 01/10/2013 - "A cell therapy-based cure of the Laron Syndrome." 01/10/2013 - "These findings indicate a novel, simply, safe and successful method for the cell therapy-based cure of the Laron Syndrome, potentially applicable to humans."
5.	Heterologous Transplantation (Xenotransplantation) 01/10/2013 - "Reversal of experimental Laron Syndrome by xenotransplantation of microencapsulated porcine Sertoli cells."