Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1
生存率、运动功能和运动里程碑:AVXS-101 与 Nusinersen 治疗 1 型脊髓性肌萎缩症患儿的比较
期刊:Advances in Therapy
影响因子:4
doi:10.1007/s12325-019-00923-8
Dabbous, Omar; Maru, Benit; Jansen, Jeroen P; Lorenzi, Maria; Cloutier, Martin; Guérin, Annie; Pivneva, Irina; Wu, Eric Q; Arjunji, Ramesh; Feltner, Douglas; Sproule, Douglas M