On Wednesday, Novartis gave a more detailed look at long-awaited clinical data that the company believes will help secure a broad approval for a successor drug to its blockbuster gene therapy ...

Nearly six years ago, the Food and Drug Administration approved Zolgensma, a Novartis gene therapy for the fatal neuron-wasting disease spinal muscular atrophy. It heralded a new era of genetic ...

Newly approved SMA therapies show promise but face global accessibility challenges, widening healthcare disparities due to high costs and limited availability. Newborn screening for SMA is ...