Keywords
Genetic therapy
Motor skills
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Abstract
Objective: To evaluate the evolution of motor function in children with Spinal Muscular Atrophy Type I, after drug infusion with the drug Zolgensma. Case details: This is a study of 2 patients, with motor function being assessed with the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders scale before medication and after 2 and 6 months. The patients were on average 2.4 ±1.69 years old, had hypotonia of the lower and upper limbs, difficulty in cervical control and in rolling and sitting movements. Final considerations: This study demonstrated that patients with muscular atrophy spinal type 1, even with low survival rates according to the literature, achieved gains on the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders scale and the acquisition of motor milestones. However, the study is limited by the intentional sample selection of patients and due to the short evaluation period, requiring work to monitor long-term results.
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