Shots:

• The FIREFISH study involves assessing of Risdiplam in infants aged 1-7 mos. with Type 1 SMA
• The P-II FIREFISH study results: met its 1EPs i.e- the proportion of infants sitting without support for at least 5sec @12mos. of treatment- assessed by the Gross Motor Scale of BSID-III- consistent safety profile with no new safety signals
• Risdiplam is an investigational SMN2 splicing modifier for SMA- currently being evaluated in 4 multicenter studies and has received US FDA’s PR status with its expected approval by 24 May- 2019

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 Ref: GlobeNewswire | Image: Roche