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

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...

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Bibliographic Details
Main Authors: Mihaela Axente, Andrada Mirea, Corina Sporea, Liliana Pădure, Cristina Manuela Drăgoi, Alina Crenguța Nicolae, Daniela Adriana Ion
Format: Article
Language:English
Published: MDPI AG 2022-09-01
Series:Pharmaceutics
Subjects:
Online Access:https://www.mdpi.com/1999-4923/14/10/2074