Roche Reports New Interim Data of JEWELFISH & RAINBOWFISH Studies for Evrysdi (risdiplam) to Treat Spinal Muscular Atrophy

 Roche Reports New Interim Data of JEWELFISH & RAINBOWFISH Studies for Evrysdi (risdiplam) to Treat Spinal Muscular Atrophy

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

Click here to­ read full press release/ article | Ref: GlobeNewswire | Image: PharmaLive

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