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Prognostication and Biomarker Potential of C26:0 Lysophosphatidylcholine in Adrenoleukodystrophy

作者:Charles J. Billington, Arpana Rayannavar, Rebecca Tryon, Tory Kaye, Ashish Gupta, Troy C. Lund, Aida Lteif, Katherine L. Adriatico, Paul J. Orchard, Bradley S. Miller, Nishitha R. Pillai · 发表于:Archives of Pediatrics and Adolescent Medicine · 年份:2025 · DOI:10.1001/jamapediatrics.2024.6774 · 被引用次数:13 · 研究领域:Peroxisome Proliferator-Activated Receptors、Metabolism and Genetic Disorders、Hyperglycemia and glycemic control in critically ill and hospitalized patients

This cohort study conducted among Minnesota children diagnosed with adrenoleukodystrophy through newborn screening examines correlation of C26:0 lysophosphatidylcholine (C26LPC) with clinical phenotype over 5 years and recommends adjusting early childhood surveillance regimens in children with lower C26LPC levels.