Gene therapy for spinal muscular atrophy (SMA): First experience with Onasemnogene abeparvovec (Zolgensma®) in a private hospital in Mexico

José Antonio Infante Cantú and Sofía Lucila Rodríguez Rivera *

Tecnologico de Monterrey. Zambrano Hellion TecSalud Hospital, Pediatric Neurology Institute, Nuevo Leon, Mexico.
 
Research Article
International Journal of Science and Research Archive, 2024 11(02), 1067–1072.
Article DOI: 10.30574/ijsra.2024.11.2.0549
 
Publication history: 
Received on 22 February 2024; revised on 30 March 2024; accepted on 02 April 2024
 
Abstract: 
Spinal muscular atrophy is a hereditary neuromuscular disease characterized by the degeneration of alpha motor neurons of the anterior horn of the spinal cord, leading to progressive symmetrical muscle weakness and a high risk for respiratory complications resulting in the need for some degree of ventilatory support.
Two infants are presented with hypotonic syndrome in which spinal muscular atrophy was diagnosed by a genetic study. Gene therapy with Zolgensma® was used to evaluate the clinical improvement in motor function according to the Chop Intend Scale.
 
Keywords: 
Spinal muscular atrophy; Genetic therapy; Motor scale; SMN Protein; Onasemnogene abeparvovec
 
Full text article in PDF: 
 
Copyright information: 

Copyright © 2024 Author(s) retain the copyright of this article. This article is published under the terms of the Creative Commons Attribution Liscense 4.0