- 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.
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