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A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1.
Topline data from Phase 3 trial expected in Q4 2024 Company to present encore 36-month efficacy, safety, and patient-reported outcomes data from the Phase 2 TOPAZ trial extension at the 2023...
New trial focuses on drug's performance in spinal muscular atrophy beyond childhood
- Tertiary endpoint data show trends of continuous improvement in activities of daily living, fatigue, and endurance over 24 months - These data indic