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Intrathecal onasemnogene abeparvovec improved HFMSE scores in patients with SMA type 2, indicating enhanced motor function. The STEER study confirmed the therapy's favorable safety profile ...

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The Phase III STEER study met its primary endpoint showing an increase from baseline in HFMSE total score in patients with SMA treated with intrathecal onasemnogene abeparvovec (OAV101 IT) OAV101 IT ...

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