First multicenter real-world analysis of switching to next-generation enzyme replacement therapies in late-onset Pompe disease
首个针对晚发型庞贝病患者改用下一代酶替代疗法的多中心真实世界分析
期刊:Journal of Neurology
影响因子:4.6
doi:10.1007/s00415-026-13689-1
Mendelsohn, Daniel H; Rosenbohm, Angela; Güttsches, Anne-Katrin; Kornblum, Cornelia; Knop, Karl Christian; Fangerau, Tanja; Nguyen-Younossi, Nam; Shahyrova, Guljan; Garcia-Angarita, Natalia; Schoser, Benedikt; Wenninger, Stephan