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Once-Daily Risdiplam Beneficial in Patients With Types 2 and 3 SMA: Lancet
Once-Daily Risdiplam is beneficial in patients with Types 2 and 3 spinal muscular atrophy (SMA), according to a recent study published in the Lancet.
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atrophy, with approval for use in patients with type 2 and type 3 spinal muscular atrophy granted on the basis of unpublished data.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 the 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.
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