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Phase 2, randomized, double-blind, placebo-controlled, nested single and multiple ascending dose study to evaluate the safety, tolerability and pharmacokinetics of ARCT-810 in adolescent and adult participants with ornithine transcarbamylase deficiency
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Treichel AJ, Finholm I, Knutson KR, Alcaino C, Whiteman ST, Brown MR, Matveyenko A, Wegner A, Kacmaz H, Mercado-Perez A, Gajdos GB, Ordog T, Grover M, Szurszewski J, Linden DR, Farrugia G, Beyder A
