New clinical trial to Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)

Roche presents its new data on the SUNFISH, JEWELFISH, RAINBOWFISH study

  • 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
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Mia

Light a candle, let it burn. 🕯 Never give up the hope, let the darkness disapear. 🌅 Had a son with X-Linked Spinal Muscular Atrophy (SMAX2)