Genentech Reports Three-Year Data of Evrysdi (risdiplam) in (FIREFISH) Study for the Treatment of Type 1 Spinal Muscular Atrophy
Shots:
- The 3yr. data from the (FIREFISH) study including 1yr. data from OLE evaluated the efficacy & safety of Evrysdi in infants aged 1-7mos. with Type 1 SMA
- The results showed improvements in survival & motor milestones, 91% of infants were alive @3yr. & the therapy continued to improve or maintain motor functions including the ability to swallow @36mos., sit without support for 30sec., stand with support & walk while holding on b/w 2 to 3yrs., no infant lost the ability after 3yrs.
- The therapy also continued to improve or maintain measures of HINE-2 b/w 24-36mos., ~50% reduction in SAEs after 12mos. & 78% b/w 1st & 3rd yr., no treatment-related AEs leading to withdrawal of treatment discontinuation
Ref: Businesswire | Image: Genentech
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