Shots:

• Roche reports the new 2-year data from Part 1 of the FIREFISH study assessing Evrysdi in infants aged 2-7mos. with symptomatic Type 1 SMA. The 2-year results in infants treated with Evrysdi showed that continued improvement and achieve motor milestones
• The exploratory analysis demonstrated 88% of infants were alive and required no permanent ventilation @2yrs.- 59% of infants were able to sit without support for at least 5sec.- 65% had maintained upright head control- 29% can turn themselves over and 30% were able to stand with support- 71% achieved a CHOP-INTEND score of ≥40 points and all infants increased their score from 12-24mos.- all infants are alive in which 100% maintained the ability to swallow and 93% were able to feed orally
• Evrysdi is an SMN2 splicing modifier- being studied in 450 patients as part of a clinical program in SMA- with patients ranging from birth to 60yrs.- and includes pre-symptomatic patients and those prior treated with other SMA-targeting therapies

­ Ref: Roche | Image: Roche