Roche Reports Results of Risdiplam in Pivotal SUNFISH Study for Patients with Type 2 or 3 Spinal Muscular Atrophy

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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