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.
Authors
- Н. Н. Волеводз (ORCID: https://orcid.org/0000-0001-6470-6318)
- Zinaida Zyuzikova (ORCID: https://orcid.org/0000-0001-6709-8231)
Institutions
- Regional Clinical Research (US)
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
- Field-Weighted Citation Impact
- 0.00