Safety and efficacy of leriglitazone in childhood cerebral adrenoleukodystrophy (NEXUS): an interim analysis of an open-label, phase 2/3 trial
利格列酮治疗儿童脑性肾上腺脑白质营养不良的安全性和有效性（NEXUS）：一项开放标签的 2/3 期试验的中期分析
期刊:EClinicalMedicine
影响因子:10
doi:10.1016/j.eclinm.2025.103265
García-Cazorla, Ángeles; Sevin, Caroline; Constante, Juliana Ribeiro; Yazbeck, Elise; Rosewich, Hendrik; Jimenez, Sandra; Chia-Yi Chiang, Gloria; Rapalino, Otto; Caruso, Paul; Balentine, Daniel; Helmer, Karl G; Bennett, Seth; Emanuele, Marco; Rodriguez-Pascau, Laura; Pizcueta, Pilar; Pina, Guillem; Vilà, Anna; Rovira, Maria; Mantilla, Adriana; Meya, Uwe; Mistry, Arun; Pascual, María; Pascual, Sílvia; Martinell, Marc; Musolino, Patricia L; Mallack, Eric
