Shots:

• The 2-year data from Part 2 of P-II/III FIREFISH study involves assessing Evrysdi in 41 infants aged 1-7mos. with symptomatic Type 1 SMA
• Results: improvement in maintained motor function b/w 12-24mos.- babies were able to sit without support for at least 5sec. after 24mos. vs 12mos. (61% vs 29%)- 92% maintained the ability to feed orally @24mos.
• The exploratory data showed 95% maintained ability to swallow- 88% infants were alive and required no permanent ventilation after 24 mos.- patients maintained upright head control (63% vs. 44%)- roll from supine to prone (44% vs 10%)- stand with support (15% vs 5%) & able to walk (4% vs 2%)- 76% achieved a CHOP-INTEND score of ≥40 @24 mos.

  Ref: Business Wire | Image: Xconomy