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

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.