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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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the ...