Risdiplam (Evrysdi): A High-Profile Drug for Treating Spinal Muscular Atrophy (SMA)
Risdiplam (Evrysdi): A High-Profile Drug for Treating Spinal Muscular Atrophy (SMA) Introduction Spinal muscular atrophy (SMA) is a rare genetic disorder that affects approximately one in 6,000 to 10,000 live births globally, causing progressive muscle weakness and atrophy. SMA is caused by mutations in the SMN1 gene, responsible for producing the survival motor neuron (SMN)…