Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA
- The Phase III STEER study showed an increase in HFMSE scores in SMA patients treated with OAV101 IT compared to sham controls.
- OAV101 IT demonstrated a favorable safety profile with similar adverse events between treatment and control groups.
- Novartis plans to share results with regulatory agencies in 2025, aiming for availability to SMA patients.
- Data will be presented at an upcoming medical meeting in 2025.
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