Roche is aiming for mid-year filings of its oral therapy for spinal muscular atrophy, risdiplam, based on promising Phase II/III data and positive regulator feedback. With a hoped-for approval in 2020, the product could add a convenient new therapy option into the SMA mix, and one that is likely to have its own impact on the treatment landscape.
SMA is the leading cause of genetic death in infants, affecting around one in 10,000 live births. The arrival of Biogen Inc
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