Summary:
- The article discusses the results of a two-year study on the use of Genentech's Evrysdi (risdiplam) in the treatment of children with spinal muscular atrophy (SMA).
- The study found that the majority of children treated with Evrysdi were able to sit, stand, and walk independently, demonstrating the drug's effectiveness in improving motor function in SMA patients.
- The data highlights the potential of Evrysdi to provide a significant improvement in the quality of life for children with SMA, a rare and debilitating genetic disorder that affects the motor neurons.