Shots:

• The RAINBOWFISH study evaluates the efficacy and safety of Evrysdi in babies aged birth to 6wks. with pre-symptomatic SMA
• The results showed that 4 out of 5 patients treated with Evrysdi for 12 mos. were able to stand & walk independently within the WHO windows for healthy children. Additionally- all five infants maintained the ability to swallow and were able to feed exclusively orally after 12mos. of treatment
• The results further support the continued clinical investigation of SRP-9001 in DMD. Additionally- the company also presents the data from the SRP-9001- 101/002/003 studies evaluating the efficacy- safety- and durability of SRP-9001 for DMD

  Ref: Roche | Image: Roche