Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
מצבים רפואיים
Spinal Muscular Atrophy (SMA)
סיכום בשפה פשוטהבעזרת בינה מלאכותית
הסיכום נכתב על־ידי מודל שפה והוא ניסיוני. המידע עלול להיות חלקי או שגוי ואינו תחליף לייעוץ רפואי.
מיקומים
1. State Institution Republican Scientific and Practical Center "Mother and Child", Minsk, Belarus
Federal State Autonomous Educational Institution of Higher Education "N.I. Pirogov Russian National Research Medical University", Ministry of Health of the Russian Federation, Moscow, Russia
Federal State Autonomous Educational Institution of Higher Education "N.I. Pirogov Russian National Research Medical University", Ministry of Health of the Russian Federation, Moscow, Russia
Federal State Autonomous Institution "National Medical Research Center for Children's Health", Ministry of Health of the Russian Federation, Moscow, Russia
Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University", Ministry of Health of the Russian Federation, Saint Petersburg, Russia
Federal State Budgetary Institution "V. A. Almazov National Medical Research Center" of the Ministry of Health of the Russian Federation, Saint Petersburg, Russia
State Autonomous Healthcare Institution of the Sverdlovsk Region "Regional Children's Clinical Hospital", Yekaterinburg, Russia
מובילי המחקר
Arina V Zinkina-Orikhan, PhD
Study Director
Director of Clinical Development Department, BIOCAD