GHR encodes growth hormone receptor (638 aa). Receptor for pituitary gland growth hormone (GH1) involved in regulating postnatal body growth. On ligand binding, couples to the JAK2/STAT5 pathway Highest expression in Adipose Subcutaneous (51.4 TPM) and Adipose Visceral Omentum (41.4 TPM).

Laron syndrome is associated with mutations in the GHR gene on chromosome 5.

The GHR protein participates in GHR internalization pathway.

GHR is classified as a druggable target (Cell Surface, Druggable Genome, and External Side Of Plasma Membrane categories) with score 10.4.

Genetic testing for GHR is available. Testing is considered confirmatory for diagnosis.

No approved treatments are currently available for Laron syndrome. An additional 2 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for Laron syndrome, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for Laron syndrome. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Phenotype severity distribution: 2 always present features, 1 very common feature.

Estimated prevalence: 1-9 in 1,000,000 (Rare).

No clinical trials have been registered for Laron syndrome.

24 publications have been identified in PubMed for Laron syndrome. Research spans Basic Science / Preclinical (25%), Epidemiology / Natural History (25%), and Review / Meta-Analysis (21%).

Recent Publications

• Guevara A (2026). [PMID: 42061055](https://pubmed.ncbi.nlm.nih.gov/42061055/). *Growth Horm IGF Res*. [Review / Meta-Analysis]

• Guevara A (2026). [PMID: 42061054](https://pubmed.ncbi.nlm.nih.gov/42061054/). *Growth Horm IGF Res*. [Basic Science / Preclinical]

• Hinrichs A (2026). [PMID: 41125144](https://pubmed.ncbi.nlm.nih.gov/41125144/). *Mol Metab*. [Basic Science / Preclinical]

• Hinrichs A (2026). [PMID: 41285399](https://pubmed.ncbi.nlm.nih.gov/41285399/). *Mol Metab*. [Other]

• Huang Z (2026). [PMID: 41781377](https://pubmed.ncbi.nlm.nih.gov/41781377/). *Int J Oral Sci*. [Review / Meta-Analysis]

• Mishra V (2026). [PMID: 41866985](https://pubmed.ncbi.nlm.nih.gov/41866985/). *J Neurotrauma*. [Other]

• Manni E (2026). [PMID: 41905220](https://pubmed.ncbi.nlm.nih.gov/41905220/). *Cytokine*. [Review / Meta-Analysis]

• Laron Z (2026). [PMID: 42053457](https://pubmed.ncbi.nlm.nih.gov/42053457/). *J Pediatr Endocrinol Metab*. [Epidemiology / Natural History]

• Uçaktürk SA (2026). [PMID: 41099230](https://pubmed.ncbi.nlm.nih.gov/41099230/). *J Pediatr Endocrinol Metab*. [Case Report / Case Series]

• Ramon-Krauel M (2026). [PMID: 40626687](https://pubmed.ncbi.nlm.nih.gov/40626687/). *J Clin Endocrinol Metab*. [Epidemiology / Natural History]