Phase 2 Study to Test Therapeutic Potential of Samelisant for Narcolepsy Type 1

Published Date: 13 Jun 2025

Phase 2 Study to Test Therapeutic Potential of Samelisant for Narcolepsy Type 1

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Real-world 12‑month results from WeSMA, a phase 4 study, showed that oral risdiplam is well tolerated in adults with SMA, with stable motor function and consistent safety findings.

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New 12-month interim data showed functional gains with BBP-418 vs placebo, consistent benefit across genotype, age, and baseline pulmonary function subgroups, and a safety profile comparable to placebo.

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