Shots:
- The company has reported the results from Part 2 of the FIREFISH study evaluating the efficacy and safety of Evrysdi in 41 infants aged 1-7 mos. with symptomatic Type 1 SMA
- The study met its 1EP i.e., 29% of infants sitting without support for at least 5 sec., 93% of infants were alive and 85% required no permanent ventilation @ 12 mos., median duration of treatment (15.2mos.) with median age was 20.7mos, 90% achieved a CHOP-INTEND
- Additionally, the study met its 2EPs i.e., 78% of infants classified as HINE-2 responders. The therapy also showed improvement in survival and motor milestones & the safety was consistent with its known safety profile
Click here to to read full press release/ article | Ref: Roche| Image: Wikipedia
The post Roche Publishes the Results of Evrysdi (risdiplam) in FIREFISH Study to Treat of Type 1 SMA at NEJM first appeared on PharmaShots.
