- Natural History of Type 1 Interferonopathies: Insights From a European Cohort — Recruiting • Neurology • NCT07040774.
- European study tracking type 1 interferonopathy progression in children and adults to improve diagnosis, prognosis, and personalise treatment options.
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Type I interferonopathies are rare autoinflammatory disorders caused by genetic defects and associated with significant morbidity and mortality. These diseases are refractory to conventional immunosuppressive therapies. They typically occur in childhood, although disease onset in adulthood has been observed. The clinical spectrum is wide and mainly involves the central nervous system. Joint involvement is also common, and more rarely, haematological features such as cytopenias or immunodeficiency may be observed. Nearly all patients show consistent over-activation of the type I IFN pathway, as evidenced, the expression of IFN-stimulated genes, the so-called…