Shots:

• The new interim data from two studies i.e JEWELFISH open-label study evaluating Evrysdi in people aged 1 to 60 yrs previously treated with another SMA-targeting therapy & RAINBOWFISH study evaluating Evrysdi in babies from birth to 6 wks. with pre-symptomatic SMA
• The JEWELFISH showed stabilization in motor function as measured by change from baseline in MFM @ 1yr. and favourable safety profile in previously treated patients with SMA-targeting therapy & an increase in SMN protein levels
• The preliminary efficacy data from RAINBOWFISH showed that infants treated for 1 yr. were able to sit- stand and walk- improvements in motor function. The data is presented at SMA 2021

Ref: GlobeNewswire | Image: Roche