Australian public assessment report for Risdiplam

Although the efficacy results obtained from Part 1 of Study BP39055 are exploratory, there is reasonably high concordance at Month 12 with the pivotal Part 2 results at Month 12 for the primary and key secondary efficacy endpoints. The results from Part 1 are both supportive of efficacy in SMA Types 2 and 3, and provided valuable long-term data (up to 2 years) to suggest that efficacy is ... ................
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