5589320 FOLLOW-UP RESULTS OF A PHASE 2 STUDY ASSESSING THE SAFETY AND EFFICACY OF MITAPIVAT TREATMENT, AN ORAL PYRUVATE KINASE ACTIVATOR, FOR UP TO 60 WEEKS IN SUBJECTS WITH SICKLE CELL DISEASE
5589320 一项评估口服丙酮酸激酶激活剂米他匹伐治疗在镰状细胞病患者中长达 60 周的安全性和有效性的 II 期研究的后续结果
期刊:PLoS Medicine
影响因子:9.9
doi:10.1371/journal.pmed.1004232
Vinther, Johan L; Cadman, Tim; Avraam, Demetris; Ekstrøm, Claus T; Sørensen, Thorkild I A; Elhakeem, Ahmed; Santos, Ana C; de Moira, Angela Pinot; Heude, Barbara; Iñiguez, Carmen; Pizzi, Costanza; Simons, Elinor; Voerman, Ellis; Corpeleijn, Eva; Zariouh, Faryal; Santorelli, Gilian; Inskip, Hazel M; Barros, Henrique; Carson, Jennie; Harris, Jennifer R; Nader, Johanna L; Ronkainen, Justiina; Strandberg-Larsen, Katrine; Santa-Marina, Loreto; Calas, Lucinda; Cederkvist, Luise; Popovic, Maja; Charles, Marie-Aline; Welten, Marieke; Vrijheid, Martine; Azad, Meghan; Subbarao, Padmaja; Burton, Paul; Mandhane, Puishkumar J; Huang, Rae-Chi; Wilson, Rebecca C; Haakma, Sido; Fernández-Barrés, Sílvia; Turvey, Stuart; Santos, Susana; Tough, Suzanne C; Sebert, Sylvain; Moraes, Theo J; Salika, Theodosia; Jaddoe, Vincent W V; Lawlor, Deborah A; Nybo Andersen, Anne-Marie; Della Pasqua, ODP; Ceci, AC; Ruggieri, LR; Oosterholt, SO; di Deo, AdD; Bonifazi, FB; van Dijk, MJ; Rab, MAE; van Oirschot, BA; Bos, JF; Derichs, C; Rijneveld, AW; Cnossen, MH; Nur, E; Biemond, BJ; Bartels, M; Jans, JJM; van Solinge, WW; Schutgens, REG; van Wijk, R; van Beers, EJ
