National Center for Advancing and Translational Sciences Genetic and Rare Diseases Information Center, a program of the National Center for Advancing and Translational Sciences

Growth hormone deficiency

Growth hormone deficiency (GHD) is characterized by abnormally short height due to lack (or shortage) of growth hormone. It can be congenital (present at birth) or acquired. Most cases are identified in children. Although it is uncommon, growth hormone deficiency may also be diagnosed in adults.[1] Too little growth hormone can cause short stature in children, and changes in muscle mass, cholesterol levels, and bone strength in adults.[2] Most of the time, no single clear cause can be identified but several genetic causes of GHD have been described, such as mutations in the to POU1F1/Pit1 , PROP1 GHRH and GH1 genes.[3] In adolescents, puberty may be delayed or absent. Treatment involves growth hormone injections.[1]

Last updated: 6/3/2016

FDA-Approved Treatments

The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.

  • Somatropin (r-DNA) for injection (Brand name: Genotropin) - Manufactured by Pfizer, Inc.
    FDA-approved indication: For long-term treatment of growth failure in children born small for gestational age who fail to manifest catch-up growth by two years of age. Also for the treatment of adults with growth hormone deficiency.
    National Library of Medicine Drug Information Portal
  • Somatropin (r-DNA) for injection (Brand name: Humatrope) - Manufactured by Eli Lilly and Company
    FDA-approved indication: For the long-term treatment of children who have growth failure due to inadequate secretion of normal endogenous growth hormone.
    National Library of Medicine Drug Information Portal
  • Macimorelin acetate (Brand name: Macrelin) - Manufactured by Strongbridge Biopharma (Note: This product is used for diagnosis and is not a medical treatment.)
    FDA-approved indication: December 2017, macimorelin acetate (Macrelin) was approved for the diagnosis of adult growth hormone deficiency (AGHD).
    National Library of Medicine Drug Information Portal
  • Somatropin (r-DNA) for injection (Brand name: Norditropin) - Manufactured by Novo Nordisk Pharmaceuticals
    FDA-approved indication: Long-term treatment of children who have growth failure due to inadequate secretion of endogenous growth hormone.
    National Library of Medicine Drug Information Portal
  • Somatropin (r-DNA) for injection (Brand name: Nutropin AQ) - Manufactured by Genentech, Inc.
    FDA-approved indication: For use in the long-term treatment of children who have growth failure due to a lack of adequate endogenous growth hormone secretion. Also for treatment of children with growth failure associated with chronic renal insufficiency and as replacement therapy for growth hormone deficiency in adults after epiphyseal closure.
    National Library of Medicine Drug Information Portal
  • Somatropin (r-DNA) for injection (Brand name: Saizen) - Manufactured by EMD Serono, Inc.
    FDA-approved indication: For the long term treatment of children with growth failure due to inadequate secretion of endogenous growth hormone. Also for the treatment of adults with GHD that started as a child or as an adult.
    National Library of Medicine Drug Information Portal

If you need medical advice, you can look for doctors or other healthcare professionals who have experience with this disease. You may find these specialists through advocacy organizations, clinical trials, or articles published in medical journals. You may also want to contact a university or tertiary medical center in your area, because these centers tend to see more complex cases and have the latest technology and treatments.

If you can’t find a specialist in your local area, try contacting national or international specialists. They may be able to refer you to someone they know through conferences or research efforts. Some specialists may be willing to consult with you or your local doctors over the phone or by email if you can't travel to them for care.

You can find more tips in our guide, How to Find a Disease Specialist. We also encourage you to explore the rest of this page to find resources that can help you find specialists.

Healthcare Resources

Research helps us better understand diseases and can lead to advances in diagnosis and treatment. This section provides resources to help you learn about medical research and ways to get involved.

Clinical Research Resources

  • ClinicalTrials.gov lists trials that are related to Growth hormone deficiency. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.

    Please note: Studies listed on the ClinicalTrials.gov website are listed for informational purposes only; being listed does not reflect an endorsement by GARD or the NIH. We strongly recommend that you talk with a trusted healthcare provider before choosing to participate in any clinical study.

Support and advocacy groups can help you connect with other patients and families, and they can provide valuable services. Many develop patient-centered information and are the driving force behind research for better treatments and possible cures. They can direct you to research, resources, and services. Many organizations also have experts who serve as medical advisors or provide lists of doctors/clinics. Visit the group’s website or contact them to learn about the services they offer. Inclusion on this list is not an endorsement by GARD.

Organizations Supporting this Disease

Organizations Providing General Support

Living with a genetic or rare disease can impact the daily lives of patients and families. These resources can help families navigate various aspects of living with a rare disease.

Financial Resources

  • Good Days provides help to patients with life-altering conditions. Assistance includes help with the cost of medications and travel.

These resources provide more information about this condition or associated symptoms. The in-depth resources contain medical and scientific language that may be hard to understand. You may want to review these resources with a medical professional.

Where to Start

  • MedlinePlus was designed by the National Library of Medicine to help you research your health questions, and it provides more information about this topic.
  • The National Organization for Rare Disorders (NORD) has a report for patients and families about this condition. NORD is a patient advocacy organization for individuals with rare diseases and the organizations that serve them.
  • The Pediatric Endocrine Society provides an online fact sheet entitled "Growth Hormone Deficiency: A Guide for Families."
  • The MAGIC Foundation provides information about this condition. The MAGIC Foundation provides support services for the families of children with a wide variety of chronic and/or critical disorders, syndromes and diseases that affect a child's growth.

In-Depth Information

Questions sent to GARD may be posted here if the information could be helpful to others. We remove all identifying information when posting a question to protect your privacy. If you do not want your question posted, please let us know. Submit a new question

  • I am having difficulty obtaining consistent US prevalence data on Growth hormone deficiency in children and Growth hormone deficiency in adults. Any information you can provide would be greatly appreciated. See answer


  1. Growth hormone deficiency - children. MedlinePlus. 2015; http://www.nlm.nih.gov/medlineplus/ency/article/001176.htm.
  2. Growth hormone test. MedlinePlus. 2014; http://www.nlm.nih.gov/medlineplus/ency/article/003706.htm.
  3. Richmond EJ & Rogol AD. Diagnosis of growth hormone deficiency in children. UpToDate. May 2016; http://www.uptodate.com/contents/diagnosis-of-growth-hormone-deficiency-in-children.