Risdiplam is an orally bioavailable mRNA splicing modifier being investigated for the treatment of spinal muscular atrophy (SMA). It increases systemic SMN protein concentrations by improving the efficiency of SMN2 gene transcription. This mechanism of action is similar to its predecessor [nusinersen], the biggest difference being their route of administration: nusinersen requires intrathecal administration, whereas risdiplam offers the ease of oral bioavailability.

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