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Severe combined immunodeficiency due to CTPS1 deficiency

ORPHA:420573· ICD-10 D81.2

Definition

A rare primary immunodeficiency disorder due to impaired capacity of activated T- and B-cells to proliferate in response to antigen receptor-mediated activation characterized by early-onset, severe, persistent and/or recurrent viral infections due to Epstein-Barr virus (EBV) and Varicella Zoster virus (VZV, including generalized varicella), as well as recurrent sino-pulmonary bacterial infections due to encapsulated pathogens.

Prevalence
<1 / 1 000 000
Inheritance
Autosomal recessive
Age of onset
Childhood, Infancy, Neonatal