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Hoyeraal Hreidarsson syndrome MeSH Supplementary Concept Data 2025
A severe variant of DKC that is characterized by multisystem involvement and early onset in utero. Patients with HHS show intrauterine growth retardation; MICROCEPHALY; DEVELOPMENTAL DISABILITIES, and bone marrow failure resulting in immunodeficiency, cerebellar hypoplasia, and sometimes enteropathy. Death often occurs in childhood. Caused by mutations in the DKC1 gene. OMIM: 305000