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Roche Presents New 2-year Data For Evrysdi (risdiplam) In Infants With Type 1 Spinal Muscular Atrophy (SMA)

Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)

Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)

  • Exploratory efficacy data showed 88% of infants treated with Evrysdi were alive and did not require permanent ventilation at two years
  • 59% of infants were able to sit without support for at least 5 seconds
  • No new safety signals were identified
  • In August, the FDA approved Evrysdi for the treatment of SMA in adults and children 2 months and older

For the full text by Roche please click here.

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