Shots:

• The approval is based on two studies i.e. FIREFISH & SUNFISH. The FIREFISH study involves assessing Evrysdi in infants aged 2-7mos. with symptomatic Type 1 SMA- and the SUNFISH study assesses Evrysdi in children & young adults aged 2-25yrs. with Type 2 or 3 SMA
• Evrysdi demonstrated improvement in motor function- 29.3% of participants were able to sit without support for at least 5sec.- 85.4% of participants shows event-free survival without permanent ventilation after 12 mos. of treatment
• Evrysdi is a pre-mRNA splicing modifier of SMN2 designed to treat SMA in patients aged ≥2mos. and is the first at-home administered treatment for SMA

  Ref: Newswire Canada | Image: Roche