ArticleArquivos de neuro-psiquiatria2026
Nusinersen for type-III spinal muscular atrophy: a 12-month retrospective study in a Brazilian cohort.
Article in Arquivos de neuro-psiquiatria, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Spinal muscular atrophy (SMA) is a progressive, autosomal recessive motor neuron disorder caused by mutations in the Objective: The present study investigated treatment responses in SMA type-III patients subjected to nusinersen therapy. Methods: Pediatric and adult patients with genetically confirmed 5q SMA type III on nusinersen treatment for at least 12 months were included in the current retrospective study. We collected data on demographics, genetics, nutritional status, swallowing function, and adverse events associated with nusinersen treatment. Motor function was assessed in all patients using the Hammersmith Functional Motor Scale-Expanded (HFMSE), and respiratory function was evaluated through forced vital capacity in patients aged 6 and older. Results: Nineteen patients (12 males; 63.2%) were included in the study. Most patients (94.7%) had a homozygous Conclusion: Nusinersen was well tolerated and improved motor function in SMA type-III patients, supporting its potential benefits in real-world settings.
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