Shots:

• The pivotal part 2 SUNFISH study involves assessing Risdiplam vs PBO in patients aged 2-25 yrs. with type 2 or 3 spinal muscular atrophy
• The SUNFISH study resulted in meeting its 1EPs i.e- change in the Motor Function Measure 32 (MFM-32) scale @12mos. with consistent safety profile and no new signals were identified
• Risdiplam is an investigational SMN2 splicing modifier- designed to increase and sustain SMN protein levels- both in CNS and peripheral tissues of the body- being evaluated in patients ranging from birth to 60 yrs. old including patients prior treated with SMA targeting therapies

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Ref: Globe Newswire | Image: Roche