Studies in the Area of Aicardi Goutieres Syndrome Reported from Children's Hospital Philadelphia (Systemic Complications of Aicardi Goutieres Syndrome Using Real-world Data).

Předmět:	AICARDI syndrome MOLECULAR genetics CLINICAL trials monitoring CHILDREN'S hospitals NEUROLOGICAL disorders
Zdroj:	Clinical Trials Week; 10/31/2024, p1929-1929, 1p
Abstrakt:	A report from Children's Hospital Philadelphia discusses the systemic complications of Aicardi Goutieres Syndrome (AGS), a rare genetic interferonopathy with diverse multisystemic effects. The study used real-world data from medical records to characterize the disease burden in 167 individuals with genetically confirmed AGS. The research highlights the heterogeneity of AGS, emphasizing the impact of systemic symptoms on neurological outcomes and the need for evidence-based clinical monitoring guidelines and clinical trial design. [Extracted from the article]
Databáze:	Complementary Index
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