Risdiplam is primarily used in the treatment of spinal muscular atrophy (SMA), a genetic disorder characterized by muscle weakness and atrophy due to the loss of motor neurons. It works by modifying the splicing of the SMN2 gene, increasing the production of survival motor neuron (SMN) protein, which is crucial for motor neuron function. This oral medication offers a convenient treatment option, especially for patients who may have difficulty with other forms of therapy like injections or infusions. It is approved for use in adults, children, and infants, providing a broad therapeutic scope. Its ability to be administered at home enhances patient compliance and quality of life. Ongoing research continues to explore its long-term efficacy and potential applications in other neuromuscular disorders.