The firm at the JP Morgan Healthcare Conference additionally expects a regulatory decision on its gene therapy RGX-121 for Hunter syndrome next month.
Positive interim efficacy data in 2025 underscore potential of ETX101 as a one-time, disease-modifying medicine for Dravet syndrome –– Internal GMP manufacturing capable of supporting pivotal trial ...
Ruyi Ding (Northeastern University), Tong Zhou (Northeastern University), Lili Su (Northeastern University), Aidong Adam Ding (Northeastern University), Xiaolin Xu (Northeastern University), Yunsi Fei ...