Novartis receives European Commission approval for Itvisma® for spinal muscular atrophy (SMA)
Novartis receives European Commission approval for Itvisma® for spinal muscular atrophy (SMA)
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- First gene replacement therapy in the EU for broad population with SMA, including children two years and older, teens and adults
- Fixed, one-time dose designed to replace the faulty SMN1 gene, providing a distinct option from ongoing dosing approaches