Reported Earlier, Novartis Reports Topline Results From Phase III STEER Study; Study Met Its Primary Endpoint
Reported Earlier, Novartis Reports Topline Results From Phase III STEER Study; Study Met Its Primary Endpoint
早前報道，諾華公司公佈了第三階段STEER研究的頂部結果；該研究達到了其主要終點。
This pivotal study assessed the efficacy and safety of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to less than 18 years who are able to sit but have never walked independently.
本研究評估了在未治療的2型脊髓性肌萎縮症（SMA）患者中使用實驗性脊髓內注射onasemnogene abeparvovec（OAV101 IT）的療效和安全性，患者年齡在2歲至18歲之間，能夠坐但從未獨立行走過。
Efficacy and safety results for OAV101 IT were compared against a sham control, a procedure designed to mimic the administration of an investigational drug, without delivering any active treatment.
OAV101 IT的療效和安全性結果與假操作對照組進行比較，該程序旨在模擬實驗藥物的給藥，但不提供任何有效治療。