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PubMed This is a summary of 20 peer-reviewed journal articles Updated
Endocrinology

Growth, Hormones, and Your Child’s Metabolism

At a Glance

Children with Mulibrey nanism experience profound growth failure and typically reach an adult height of 4 to 5 feet. Growth hormone therapy is generally ineffective and carries severe cardiac and tumor risks. Patients require lifelong monitoring for type 2 diabetes, fatty liver disease, and premature ovarian insufficiency.

Growth and metabolic health are central to the journey of a child with Mulibrey nanism. Because the TRIM37 gene acts as a manager for how cells grow and process energy, its absence leads to a specific pattern of physical development and long-term metabolic risks [1][2].

Understanding Growth Failure and Expectations

Children with Mulibrey nanism experience what is called profound growth failure, which typically begins before birth (prenatal onset) and continues throughout childhood [1][3].

This happens because the lack of TRIM37 protein disrupts several growth processes:

  • Bone Growth: In the growth plates of long bones, cells called chondrocytes cannot multiply as quickly as they should [4][5].
  • Cellular Signaling: A pathway called mTORC1, which acts like a “green light” for cell growth, is less active [2].
  • Cell Division: Errors in how cells divide can lead to a lower total number of cells in the body’s tissues [6][7].

As a parent planning for your child’s future, it helps to have realistic expectations. Children with this condition will be significantly shorter than their peers. Adult height typically falls between 4 and 5 feet (approximately 135 to 150 cm).

The Truth About Growth Hormone Therapy

Many parents understandably ask about Growth Hormone (GH) therapy to help their child gain height. However, standard medical literature indicates that GH therapy is generally ineffective for the profound, prenatal-onset growth failure specific to Mulibrey nanism [8][9].

Furthermore, pursuing GH therapy in this condition carries significant risks:

  • Tumor Risk: Because children with Mulibrey nanism already have an increased risk of developing certain tumors (like Wilms tumor), growth-promoting hormones could potentially stimulate abnormal cell growth [10][11].
  • Cardiac Strain: GH therapy can put extra work on the heart, which is highly dangerous for a child already dealing with pericardial stiffness or restrictive cardiomyopathy [3].

Discuss this openly with your pediatric endocrinologist to avoid treatments that offer false hope while risking your child’s safety.

Metabolic Risks: Diabetes and Liver Health

As children with Mulibrey nanism grow into adolescence and adulthood, their bodies may process sugar and fats differently [1][12].

  • Insulin Resistance and Type 2 Diabetes: There is a significantly higher risk for developing Type 2 Diabetes [1][12]. This often begins with insulin resistance, where the body’s cells don’t respond well to the hormone that manages blood sugar.
  • Fatty Liver Disease: The liver can accumulate extra fat (steatosis), which can eventually lead to scarring or fibrosis [13][1].
  • Hypertension: High blood pressure is common and should be monitored regularly, as it can put additional strain on the heart and kidneys [1][14].

Reproductive Health and POI

For females with Mulibrey nanism, the TRIM37 gene’s role in cell division affects the ovaries [12][1].

  • Premature Ovarian Insufficiency (POI): Most females with the condition experience an early depletion of egg follicles [12][15]. This means the ovaries may stop working much earlier than normal, often before age 40, a condition called POI [16][17].
  • Puberty and Fertility: Puberty may be delayed or incomplete. Many girls will require Hormone Replacement Therapy (HRT) to support bone health, heart health, and the development of secondary sexual characteristics [18][19].
  • Males: Males may also face fertility challenges, including germ cell aplasia, where the cells that produce sperm are missing or reduced [12].

Managing these hormonal and metabolic factors is a lifelong process. Regular blood work to check glucose, liver enzymes, and hormone levels allows your medical team to intervene early and support your child’s overall health [1][20].

Common questions in this guide

Why is my child with Mulibrey nanism so short?
Children with Mulibrey nanism experience profound growth failure due to a missing TRIM37 protein. This disrupts normal bone growth, cell division, and cellular signaling, typically resulting in an adult height between 4 and 5 feet.
Can growth hormone therapy help a child with Mulibrey nanism gain height?
Growth hormone therapy is generally ineffective for the specific type of prenatal-onset growth failure seen in Mulibrey nanism. More importantly, it is considered dangerous because it can dangerously strain the heart and increase the risk of certain tumors.
What metabolic conditions should we watch for as our child grows?
As children with this condition grow, they face an increased risk of insulin resistance, type 2 diabetes, fatty liver disease, and high blood pressure. Regular blood work and monitoring are essential to manage these lifelong risks.
How does Mulibrey nanism affect puberty and reproductive health in girls?
Many females with the condition experience delayed puberty and premature ovarian insufficiency (POI), meaning the ovaries may stop working early in life. Pediatric gynecologists often prescribe hormone replacement therapy (HRT) to support bone health, heart health, and normal development.

Questions to Ask Your Doctor

Curated prompts to bring to your next appointment.

  1. 1.What are the specific cardiac and oncological risks associated with Growth Hormone therapy in our child's case?
  2. 2.Should my daughter see a pediatric gynecologist early to monitor for signs of premature ovarian insufficiency?
  3. 3.How often should we screen for insulin resistance or type 2 diabetes?
  4. 4.Is the fatty liver disease we see in Mulibrey nanism managed differently than in other conditions?

Questions For You

Tap a prompt to share your answer — we'll use it plus this page's context to start a tailored conversation.

References

References (20)
  1. 1

    TRIMming down to TRIM37: Relevance to Inflammation, Cardiovascular Disorders, and Cancer in MULIBREY Nanism.

    Brigant B, Metzinger-Le Meuth V, Rochette J, Metzinger L

    International journal of molecular sciences 2018; (20(1)) doi:10.3390/ijms20010067.

    PMID: 30586926
  2. 2

    TRIM37 deficiency induces autophagy through deregulating the MTORC1-TFEB axis.

    Wang W, Xia Z, Farré JC, Subramani S

    Autophagy 2018; (14(9)):1574-1585 doi:10.1080/15548627.2018.1463120.

    PMID: 29940807
  3. 3

    The Importance of Early Pericardiectomy in Mulibrey Nanism Syndrome, a Case Report.

    Cordova Sanchez A, Vasigh M, Carhart R

    Journal of investigative medicine high impact case reports 2022; (10()):23247096221077816 doi:10.1177/23247096221077816.

    PMID: 35257621
  4. 4

    A proteomic study of the downregulation of TRIM37 on chondrocytes: Implications for the MULIBREY syndrome.

    Brigant B, Metzinger-Le Meuth V, Boyartchuk V, et al.

    Bone 2024; (187()):117205 doi:10.1016/j.bone.2024.117205.

    PMID: 39019132
  5. 5

    TRIM37 is highly expressed during mitosis in CHON-002 chondrocytes cell line and is regulated by miR-223.

    Brigant B, Demont Y, Ouled-Haddou H, et al.

    Bone 2020; (137()):115393 doi:10.1016/j.bone.2020.115393.

    PMID: 32353567
  6. 6

    Mosaic variegated aneuploidy as a novel feature in patients with Mulibrey nanism and TRIM37 variants.

    Hakonen AH, Karlberg S, Kiiski K, et al.

    Journal of medical genetics 2026; (63(5)):308-311 doi:10.1136/jmg-2025-111325.

    PMID: 41702694
  7. 7

    TRIM37: a critical orchestrator of centrosome function.

    Domínguez-Calvo A, Gönczy P, Holland AJ, Balestra FR

    Cell cycle (Georgetown, Tex.) 2021; (20(23)):2443-2451 doi:10.1080/15384101.2021.1988289.

    PMID: 34672905
  8. 8

    Clinical Indications for Growth Hormone Therapy.

    Danowitz M, Grimberg A

    Advances in pediatrics 2022; (69(1)):203-217 doi:10.1016/j.yapd.2022.03.005.

    PMID: 35985710
  9. 9

    Noonan Syndrome, Cancer Risk, and Growth Hormone Treatment

    Demir K, Yüksek Acınıklı K

    Journal of clinical research in pediatric endocrinology 2025; (17(4)):370-378 doi:10.4274/jcrpe.galenos.2025.2024-9-13.

    PMID: 39974721
  10. 10

    Safety of growth hormone replacement in survivors of cancer and intracranial and pituitary tumours: a consensus statement.

    Boguszewski MCS, Boguszewski CL, Chemaitilly W, et al.

    European journal of endocrinology 2022; (186(6)):P35-P52 doi:10.1530/EJE-21-1186.

    PMID: 35319491
  11. 11

    Growth Hormone Deficiency and Treatment in Childhood Cancer Survivors.

    Pollock NI, Cohen LE

    Frontiers in endocrinology 2021; (12()):745932 doi:10.3389/fendo.2021.745932.

    PMID: 34745010
  12. 12

    Trim37-deficient mice recapitulate several features of the multi-organ disorder Mulibrey nanism.

    Kettunen KM, Karikoski R, Hämäläinen RH, et al.

    Biology open 2016; (5(5)):584-95 doi:10.1242/bio.016246.

    PMID: 27044324
  13. 13

    Liver pathology and biochemistry in patients with mutations in TRIM37 gene (Mulibrey nanism).

    Sivunen J, Karlberg S, Kivisaari R, et al.

    Liver international : official journal of the International Association for the Study of the Liver 2022; (42(6)):1369-1378 doi:10.1111/liv.15213.

    PMID: 35220664
  14. 14

    Renal findings in patients with Mulibrey nanism.

    Sivunen J, Karlberg S, Lohi J, et al.

    Pediatric nephrology (Berlin, Germany) 2017; (32(9)):1531-1536 doi:10.1007/s00467-017-3669-5.

    PMID: 28432469
  15. 15

    Premature ovarian insufficiency and early depletion of the ovarian reserve in the monogenic Mulibrey nanism disorder.

    Karlberg S, Tiitinen A, Alfthan H, Lipsanen-Nyman M

    Human reproduction (Oxford, England) 2018; (33(7)):1254-1261 doi:10.1093/humrep/dey103.

    PMID: 29860321
  16. 16

    Autoimmune Diseases in Patients with Premature Ovarian Insufficiency-Our Current State of Knowledge.

    Szeliga A, Calik-Ksepka A, Maciejewska-Jeske M, et al.

    International journal of molecular sciences 2021; (22(5)) doi:10.3390/ijms22052594.

    PMID: 33807517
  17. 17

    Reproductive health in Turner's syndrome: from puberty to pregnancy.

    Porcu E, Cipriani L, Damiano G

    Frontiers in endocrinology 2023; (14()):1269009 doi:10.3389/fendo.2023.1269009.

    PMID: 38116311
  18. 18

    Premature and Early Menopause in Relation to Cardiovascular Disease.

    Schipper I, Louwers YV

    Seminars in reproductive medicine 2020; (38(4-05)):270-276 doi:10.1055/s-0040-1722318.

    PMID: 33511582
  19. 19

    Transdermal versus oral hormone therapy in premature ovarian insufficiency.

    Beitl K, Widmann K, Marculescu R, et al.

    Menopause (New York, N.Y.) 2025; (32(10)):913-919 doi:10.1097/GME.0000000000002588.

    PMID: 40627723
  20. 20

    Wilms tumor with Mulibrey Nanism: A case report and review of literature.

    Upasana K, Thakkar D, Gautam D, et al.

    Cancer reports (Hoboken, N.J.) 2022; (5(5)):e1512 doi:10.1002/cnr2.1512.

    PMID: 34309235

This page is for informational purposes only and does not replace professional medical advice. Always consult your pediatric endocrinologist before considering any hormonal therapies for your child.

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