Genentech Presents Results of Two-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA at AAN2021

 Genentech Presents Results of Two-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA at AAN2021

Genentech Presents Results of Two-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA at AAN2021

Shots:

  • The 2-year data from Part 2 of P-II/III FIREFISH study involves assessing Evrysdi in 41 infants aged 1-7mos. with symptomatic Type 1 SMA
  • Results: improvement in maintained motor function b/w 12-24mos., babies were able to sit without support for at least 5sec. after 24mos. vs 12mos. (61% vs 29%), 92% maintained the ability to feed orally @24mos.
  • The exploratory data showed 95% maintained ability to swallow, 88% infants were alive and required no permanent ventilation after 24 mos., patients maintained upright head control (63% vs. 44%), roll from supine to prone (44% vs 10%), stand with support (15% vs 5%) & able to walk (4% vs 2%), 76% achieved a CHOP-INTEND score of ≥40 @24 mos.

Click here to­ read full press release/ article | Ref: Business Wire | Image: Xconomy

Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

Related post