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 is the first investigational gene therapy to provide clinical benefit in treatment-naïve patients with SMA aged two and older with a positive risk benefit profile
Novartis plans to share results with regulatory agencies in 2025 with the aim to make OAV101 IT available to help patients with SMA in need
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