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Reported Earlier, Novartis Reports Topline Results From Phase III STEER Study; Study Met Its Primary Endpoint

Benzinga ·  Dec 30, 2024 02:23

This pivotal study assessed the efficacy and safety of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to less than 18 years who are able to sit but have never walked independently.

Efficacy and safety results for OAV101 IT were compared against a sham control, a procedure designed to mimic the administration of an investigational drug, without delivering any active treatment.

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