Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
Titel:
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
Auteur:
Finkel, Richard S. Darras, Basil T. Mendell, Jerry R. Day, John W. Kuntz, Nancy L. Connolly, Anne M. Zaidman, Craig M. Crawford, Thomas O. Butterfield, Russell J. Shieh, Perry B. Tennekoon, Gihan Brandsema, John F. Iannaccone, Susan T. Shoffner, John Kavanagh, Sarah Macek, Thomas A. Tauscher-Wisniewski, Sitra