O09: Sepiapterin responsiveness over 14 days in children and adults with phenylketonuria: Pooled results from three phase 3 clinical trials

Genetics in Medicine Open Pub Date : 2026-01-01 Epub Date: 2026-03-05 DOI:10.1016/j.gimo.2026.104294
Melissa Lah , Nicola Longo , Maria Giżewska , Janet Thomas , Amaya Bélanger-Quintana , Ida Vanessa Schwartz , Heidi Peters , Kimberly Ingalls , Zhenming Zhao , Neil Smith , Ania Muntau
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2009:苯丙酮尿症儿童和成人患者14天以上的头孢菌素反应性:来自三个3期临床试验的汇总结果
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