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Presymptomatic Risdiplam Treatment Boosts Motor Milestones in Newborns, Secures FDA Label Extension

Published RAINBOWFISH findings underpin broader authorization for infants under two months, prompting exploration of prenatal intervention

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Overview

  • The international phase 2 RAINBOWFISH study enrolled 23 presymptomatic newborns with spinal muscular atrophy who began daily risdiplam by 16 days of age and were followed for two years.
  • Across the severity spectrum, seven of eight infants predisposed to severe SMA type 1 achieved independent sitting by 12 months and five walked by age two, while all infants with milder genotypes reached sitting and walking milestones comparable to typically developing peers.
  • No major treatment-related adverse events were observed, indicating a favorable safety profile for early risdiplam administration in newborns.
  • RAINBOWFISH outcomes prompted the FDA to extend risdiplam’s label to include infants younger than two months, shifting clinical practice toward earlier intervention.
  • Researchers are now pursuing prenatal dosing and longer-term follow-up studies under Roche sponsorship to assess durability and optimize treatment timing.