Novartis的基因治疗显示,在改善SMA 2型儿童运动功能方面有希望。 Novartis' gene therapy shows promise in improving motor functions for children with SMA Type 2.

Novartis的第三阶段STEER研究表明,在用SMA Type 2 治疗儿童和青少年方面,Calal onasemnogene abeparvovec(OAV101 IT)取得了积极的成果,这是这一群体基因治疗的首例。 Novartis' Phase III STEER study showed positive results for intrathecal onasemnogene abeparvovec (OAV101 IT) in treating children and young adults with SMA Type 2, marking a first for gene therapy in this group. 研究发现,运动功能分数有所改进,安全情况良好,具有常见副作用,包括上呼吸道感染和呕吐。 The study found improved motor function scores and a favorable safety profile, with common side effects including upper respiratory infections and vomiting. Novartis的目标是在2025年与监管机构分享这些调查结果,以便提供治疗。 Novartis aims to share these findings with regulatory agencies in 2025 to make the treatment available.