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growth hormone insensitivity syndrome (GHIS) - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

growth hormone insensitivity syndrome

MONDO:0015892

Also known as:GHISGrowth hormone insensitivity syndromesshort stature due to a defect in growth hormone receptor or post-receptor pathway

Overview

Growth hormone insensitivity syndrome (GHIS) is a group of diseases characterized by marked short stature associated with normal or elevated growth hormone (GH) concentrations, which fail to respond to exogenous GH administration. GHIS comprises growth delay due to IGF-1 deficiency, growth delay due to IGF-1 resistance, Laron syndrome, short stature due to STAT5b deficiency and primary acid-labile subunit (ALS) deficiency.

OnsetINFANTILE

Treatments1 FDA approved

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

1 FDA-approved treatment is available for growth hormone insensitivity syndrome, including MECASERMIN (INCRELEX, approved 2005).

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

No clinical trials have been registered for growth hormone insensitivity syndrome.

26 publications have been identified in PubMed for growth hormone insensitivity syndrome. Research spans Basic Science / Preclinical (42%), Case Report / Case Series (27%), and Review / Meta-Analysis (23%).

Research Type	Count	% of Total
Laboratory research	11	42%

Data assembled from 4 of 12 sources · Last updated Sep 20, 2026, 3:07 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:181393

GARD

Genetic and Rare Diseases Info Center

GARD:3924

Questions for Your Care Team

→What are the benefits and potential side effects of INCRELEX for managing growth hormone insensitivity syndrome?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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Kisho

growth hormone insensitivity syndrome

Overview

Treatment

Approved Therapies

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

LARON SYNDROME. (Growth Hormone Insensitivity) Geographical distribution and relationship to the genetic defects in the growth hormone receptor gene.

growth hormone insensitivity syndrome

Overview

Treatment

Approved Therapies

Research Landscape

Medical References

External Resources

Orphanet

GARD

Subtypes

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

LARON SYNDROME. (Growth Hormone Insensitivity) Geographical distribution and relationship to the genetic defects in the growth hormone receptor gene.