BASEL (dpa-AFX) - Novartis AG (NVS) Monday reported positive results from Phase III STEER study evaluating intrathecal onasemnogene abeparvovec to treat spinal muscular atrophy (SMA) Type 2 in individuals aged 2 to 18 years.
The study met its primary goal showing an increase from baseline across the study population in total Hammersmith Functional Motor Scale - Expanded (HFMSE) scores. HFMSE is a gold standard for SMA-specific assessment of motor ability and disease progression.
Novartis plans to share the results with regulatory agencies in 2025.
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