3-M syndrome: endocrinologist's view of a rare genetic disease. Case report

3-M syndrome is a rare hereditary disease with an autosomal recessive type of inheritance, characterized by intrauterine and severe postnatal growth retardation, phenotypic and radiological features with no intellectual disability. A little more than 200 cases of this syndrome have been described in the world. 3-M syndrome is the only disease to date that is associated with pathogenic biallelic variants in the genes CCDC8, CUL7, OBSL1. Short stature is the main clinical manifestation of 3-M syndrome. Until now, the issue of treating short stature with growth hormone remains controversial due to the small number of patients with this disease and the lack of large-scale clinical studies.

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Publication Details

Journal
Pediatrics Consilium Medicum
Published
2025-10-14
DOI
https://doi.org/10.26442/26586630.2025.3.203396
Primary Topic
Ubiquitin and proteasome pathways
Type
article
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article

3-M syndrome: endocrinologist's view of a rare genetic disease. Case report

Н. Н. Волеводз, Zinaida Zyuzikova
Pediatrics Consilium Medicum
Ubiquitin and proteasome pathways
article

3-M syndrome: endocrinologist's view of a rare genetic disease. Case report

Н. Н. Волеводз, Zinaida Zyuzikova
article en

Abstract

3-M syndrome is a rare hereditary disease with an autosomal recessive type of inheritance, characterized by intrauterine and severe postnatal growth retardation, phenotypic and radiological features with no intellectual disability. A little more than 200 cases of this syndrome have been described in the world. 3-M syndrome is the only disease to date that is associated with pathogenic biallelic variants in the genes CCDC8, CUL7, OBSL1. Short stature is the main clinical manifestation of 3-M syndrome. Until now, the issue of treating short stature with growth hormone remains controversial due to the small number of patients with this disease and the lack of large-scale clinical studies.

Pediatrics Consilium Medicum(3)
Regional Clinical Research (US)
Openalex Percentile: Top 74%
Ubiquitin and proteasome pathways
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