Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment.

Katlyn E McGrattan,Richard D Shell,Rebecca Hurst-Davis,Sally Dunaway Young, Eamonn O'Brien,Arseniy Lavrov,Shiri Wallach,Nicole LaMarca,Sandra P Reyna,Basil T Darras

Journal of neuromuscular diseases（2023）

引用 1|浏览10

暂无评分

摘要

In these three clinical trials, patients with SMA type 1 who received onasemnogene abeparvovec achieved and maintained the bulbar function criteria utilized within this investigation.

查看译文

关键词

Airway protection,aspiration,bulbar function,dysphagia,gene therapy,onasemnogene abeparvovec,pneumonia,spinal muscular atrophy,swallowing,verbal communication

AI 理解论文

溯源树

样例

生成溯源树，研究论文发展脉络

Chat Paper

正在生成论文摘要