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)
Conditions
Spinal Muscular Atrophy (SMA)
Simple summaryAI-assisted
This summary is produced by a language model and is experimental. It may be incomplete or inaccurate and is not a substitute for professional medical advice.
Locations
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
Study leads
Arina V Zinkina-Orikhan, PhD
Study Director
Director of Clinical Development Department, BIOCAD