Dataset from Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
收藏NIAID Data Ecosystem2026-05-10 收录
下载链接：
https://doi.org/10.25934/PR00012298
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资源简介：
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
创建时间：
2025-12-16
