Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial
三碘甲状腺素类似物 Triac 治疗 MCT8 缺乏症儿童和成人的有效性和安全性:一项国际性、单臂、开放标签的 II 期试验
期刊:Lancet Diabetes & Endocrinology
影响因子:41.8
doi:10.1016/S2213-8587(19)30155-X
Groeneweg, Stefan; Peeters, Robin P; Moran, Carla; Stoupa, Athanasia; Auriol, Françoise; Tonduti, Davide; Dica, Alice; Paone, Laura; Rozenkova, Klara; Malikova, Jana; van der Walt, Adri; de Coo, Irenaeus F M; McGowan, Anne; Lyons, Greta; Aarsen, Femke K; Barca, Diana; van Beynum, Ingrid M; van der Knoop, Marieke M; Jansen, Jurgen; Manshande, Martien; Lunsing, Roelineke J; Nowak, Stan; den Uil, Corstiaan A; Zillikens, M Carola; Visser, Frank E; Vrijmoeth, Paul; de Wit, Marie Claire Y; Wolf, Nicole I; Zandstra, Angelique; Ambegaonkar, Gautam; Singh, Yogen; de Rijke, Yolanda B; Medici, Marco; Bertini, Enrico S; Depoorter, Sylvia; Lebl, Jan; Cappa, Marco; De Meirleir, Linda; Krude, Heiko; Craiu, Dana; Zibordi, Federica; Oliver Petit, Isabelle; Polak, Michel; Chatterjee, Krishna; Visser, Theo J; Visser, W Edward