Pediatric Neurologist, Tecnologico de Monterrey. Zambrano Hellion TecSalud Hospital.
International Journal of Science and Research Archive, 2025, 16(02), 863-870
Article DOI: 10.30574/ijsra.2025.16.2.2381
Received on 06 July 2025; revised on 10 August; accepted on 13 August 2025
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® with subsequent combination treatment with Risdiplam (Everysdi®) was used to evaluate the clinical improvement in motor function according to the Chop Intend Scale.
Spinal muscular atrophy; Genetic therapy; Motor scale; Zolgensma; Survival of Motor Neuron 1 Protein
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José Antonio Infante Cantú and Sofía Lucila Rodríguez Rivera. Combination and sequential treatments for spinal muscular atrophy: First experience with Onasemnogen abeparvovec (Zolgensma®) plus Risdiplam (Everysdi®) in a private hospital in Mexico. International Journal of Science and Research Archive, 2025, 16(02), 863-870. Article DOI: https://doi.org/10.30574/ijsra.2025.16.2.2381.
Copyright © 2025 Author(s) retain the copyright of this article. This article is published under the terms of the Creative Commons Attribution Liscense 4.0
