Work

Combination and sequential treatments for spinal muscular atrophy: First experience with Onasemnogen abeparvovec (Zolgensma®) plus Risdiplam (Everysdi®) in a private hospital in Mexico

Year: 2025

Type: article

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® with subsequent combination treatment with Risdiplam (Everysdi®) was used to evaluate the clinical improvement in motor function according to the Chop Intend Scale.

Source: Zenodo (CERN European Organization for Nuclear Research)

DOI: 10.5281/zenodo.17283705

Authors: Cantú, José Antonio Infante, Rivera, Sofía Lucila Rodríguez

Institutions: Hospital Zambrano Hellion, Tecnológico de Monterrey

Language: English

Topic: Neurogenetic and Muscular Disorders Research

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

OpenAlex ID: W7085174385

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