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Frontiers in Pediatrics|June 17, 2021
Newborn Screening for Biotinidase Deficiency. The Experience of a Regional Center in ItalyAlice Maguolo, Giulia Rodella, Alice Dianin, et al.Science Translational Medicine|January 10, 2024
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduriaSonam Gurung, Oskar Vilhelmsson Timmermand, Dany Perocheau, et al.Pageof 2