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Effects of oral sepiapterin on blood Phe concentration in a broad range of patients with phenylketonuria (APHENITY): results of an international, phase 3, randomised, double-blind, placebo-controlled trial.
Muntau AC, Longo N, Ezgu F, Schwartz IVD, Lah M, Bratkovic D, Margvelashvili L, Kiykim E, Zori R, Campistol Plana J, Bélanger-Quintana A, Lund A, Guilder L, Chakrapani A, Mungan HN, Guimas A, Cabrales Guerra IDC, MacDonald A, Ingalls K, Smith N; APHENITY study group. Muntau AC, et al. Among authors: margvelashvili l. Lancet. 2024 Oct 5;404(10460):1333-1345. doi: 10.1016/S0140-6736(24)01556-3. Lancet. 2024. PMID: 39368841 Clinical Trial.
PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: a phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study.
Bratkovic D, Margvelashvili L, Tchan MC, Nisbet J, Smith N. Bratkovic D, et al. Among authors: margvelashvili l. Metabolism. 2022 Mar;128:155116. doi: 10.1016/j.metabol.2021.155116. Epub 2021 Dec 29. Metabolism. 2022. PMID: 34973284 Free article. Clinical Trial.
Child Health Care in Georgia.
Margvelashvili L, Karseladze R, Abesadze G, Kvlividze O. Margvelashvili L, et al. J Pediatr. 2016 Oct;177S:S68-S70. doi: 10.1016/j.jpeds.2016.04.044. Epub 2016 Sep 22. J Pediatr. 2016. PMID: 27666277