rarerelated tag: SMA Type 3 (Juvenile SMA or Kugelberg-Welander Disease)

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atrophy, with approval for use in patients with type 2 and type 3 spinal muscular atrophy granted on the basis of unpublished data.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.