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Roche: positive data in spinal muscular atrophy

( - Roche has announced positive data from pivotal phase 2 of Sunfish, a study evaluating risdiplam in people aged 2 to 25 with type 2 or 3 spinal muscular atrophy (SMA).
This data will be submitted to health authorities in the world, including the FDA.

The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment with risdiplam, compared to placebo. No treatment related safety findings leading to study withdrawal have been seen in any risdiplam trial to date.

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