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Author

Dinko Vitezić

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Review Open access Sep 2026

Therapeutic advances in spinal muscular atrophy: a review of clinical, safety, and economic considerations

The clinical efficacy and real-world effectiveness of approved disease-modifying therapies, including nusinersen, onasemnogene abeparvovec, and risdiplam are summarized, underscoring significant improvements in survival, motor milestones, and quality of life, particularly with early treatment initiation.

Andrej Belančić, Elvira Meni Maria Gkrinia, Yun-Wah Lam et al. · 0 citations

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