Pharmacodynamics, Efficacy, and Safety of Intraputaminal Eladocagene Exuparvovec Administered to Pediatric Patients With Aromatic L-Amino Acid Decarboxylase Deficiency Using an MR-Compatible Cannula: 48 Weeks of Follow-Up
使用磁共振兼容套管对芳香族L-氨基酸脱羧酶缺乏症患儿进行壳核内注射Eladocagene Exuparvovec的药效学、疗效和安全性研究：48周随访
期刊:Journal of Inherited Metabolic Disease
影响因子:3.8
doi:10.1002/jimd.70151
Curry, Daniel J; Pearl, Phillip L; Stone, Scellig S D; Gilbert, Donald L; Vadivelu, Sudhakar; Ben-Zeev, Bruria; Vestal, Matthew; Zafar, Muhammad; Tai, Chun-Hwei; Chou, Sheng-Che; Zibly, Zion; Ungar, Lior; Parnes, Mered; Hull, Mariam; Emrick, Lisa; Werner, Christian; Krolick, Alexis; Penematsa, Vinay; Wang, Antonia; Rahman, Rezwanur; Golden, Lee; Chien, Yin-Hsiu; Hwu, Paul Wuh-Liang
