Short-Term Safety and Efficacy of Onasemnogene Abeparvovec in 10 Patients with Spinal Muscular Atrophy: Cohort Study

• मुख्य लेखकों: Kristina S. Nevmerzhitskaya (लेखक), Elena Yu. Sapego (लेखक), Daria A. Morozova (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: "Paediatrician" Publishers LLC, 2021-12-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: Connect to this object online.

Background. The efficacy and safety of onasemnogene abeparvovec have been demonstrated in patients with spinal muscular atrophy (SMA) in several clinical and observational studies. Gene replacement therapy results in Russian patients with SMA is not investigated yet.Objective. The aim of the study i...