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

 ­ Ref: Roche | Image: Roche