Roche Report Results of Evrysdi (risdiplam) in FIREFISH Study for Infants with Type 1 SMA

 Roche Report Results of Evrysdi (risdiplam) in FIREFISH Study for Infants with Type 1 SMA

Roche Report Results of Evrysdi (risdiplam) in FIREFISH Study for Infants with Type 1 SMA

Shots:

  • Roche has reported the data from the dose-finding Part 1 of the FIREFISH study assessing Evrysdi in patients aged 1-7mos. with Type 1 SMA. Part 1 evaluated several doses of Evrysdi and determined the therapeutic dose of 0.2 mg/kg for Part 2
  • Part 1 data demonstrated that 99% of infants survive without permanent ventilation @12 mos. & 33% sit without support for at least 5sec. In an exploratory analysis, 41% received high dose, 53% achieved upright head control, and 6% were able to stand as assessed by the HINE-2., 86% were able to feed orally
  • The researcher also assessed motor function with CHOP-INTEND & demonstrated that 52% achieved a CHOP-INTEND score of ≥40 points

Click here ­to­ read full press release/ article | Ref: Roche | Image: Forbes

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