Article
Systemic complications of Aicardi Goutières syndrome using real-world data.
Molecular genetics and metabolism - 1 Jan 2000
Peixoto de Barcelos Isabella, Jan Amanda K, Modesti Nicholson, Woidill Sarah, Gavazzi Francesco, Isaacs David, D'Aiello Russell, Sevagamoorthy Anjana, Charlton Lauren, Pizzino Amy, Schmidt Johanna, van Haren Keith, Keller Stephanie, Eichler Florian, Emrick Lisa T, Fraser Jamie L, Shults Justine, Vanderver Adeline, Adang Laura A
Abstract excerpt
OBJECTIVE: Aicardi Goutières Syndrome (AGS) is a rare genetic interferonopathy associated with diverse multisystemic complications. A critical gap exists in our understanding of its longitudinal, systemic disease burden, complicated by delayed diagnosis. To address this need, real-world data extracted from existing medical records were used to characterize the longitudinal disease burden. METHODS: All subjects...
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