Spinal muscular atrophy (SMA) is the most common genetic cause of infant death. No disease-modifying treatments are available for SMA, but the increasingly precise understanding of SMA aetiopathogenesis has led to the development of promising therapeutic strategies. Here, Farevelli et al. provide an overview of established data and novel insights into SMA pathogenesis, and discuss preclinical evidence and recent advances from ongoing clinical trials.
- Irene Faravelli
- Monica Nizzardo
- Stefania Corti