Clinical and Electrophysiological Changes in Pediatric Spinal Muscular Atrophy after 2 Years of Nusinersen Treatment

• 主要な著者: Mihaela Axente (著者), Andrada Mirea (著者), Corina Sporea (著者), Liliana Pădure (著者), Cristina Manuela Drăgoi (著者), Alina Crenguța Nicolae (著者), Daniela Adriana Ion (著者)
• フォーマット: 図書
• 出版事項: MDPI AG, 2022-09-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: Connect to this object online.

In the new therapeutic era, disease-modifying treatment (nusinersen) has changed the natural evolution of spinal muscular atrophy (SMA), creating new phenotypes. The main purpose of the retrospective observational study was to explore changes in clinical evolution and electrophysiological data after...