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Evrysdi (risdiplam) is an approved treatment for spinal muscular atrophy (SMA) in patients from birth, administered daily at home as a liquid by mouth or feeding tube. It works as an SMN2 mRNA splicing modifier to increase and sustain SMN protein production in the central nervous system and peripheral tissues.
AI named Risdiplam (Evrysdi) from October 2025 to December 2025.