Novartis is on the final straight to approval of its spinal muscular atrophy (SMA) gene therapy Zolgensma in the EU, after getting a recommendation for approval from the CHMP. The positive opinion ...

Novartis is set to launch its one-off gene therapy Zolgensma for the ultra-rare muscle-wasting disease spinal muscular atrophy (SMA) in Germany at the start of July priced at 1,945,000 euros, and ...

Treatment with salanersen slowed neurodegeneration and improved motor function in children with SMA in a Phase 1 trial, ...