Lancet neurology
The most recent articles from:
Lancet Neurol
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Risdiplam is an oral small molecule approved for the treatment of patients with spinal muscular atrophy, with approval for use in patients with type 2 and type 3 spinal muscular atrophy granted on the basis of unpublished data. The drug modifies pre-mRNA splicing of the SMN2 gene to increase production of functional SMN. We aimed to investigate the safety and efficacy of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy. ⋯ F Hoffmann-La Roche.
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Historical Article
Neuroscience in the 21st century: circuits, computation, and behaviour.