Types And Treatments For Spinal Muscular Atrophy

Gene Therapy

Since the cause of spinal muscular atrophy has been discovered, gene therapy has become an option. Researchers know exactly which gene mutations lead to the development of symptoms. With gene therapy, a patient is given a one-time treatment replacing their missing or defective SMN1 gene. They then have a working copy of the gene and may be able to restore some of their motor function. The gene is responsible for creating adequate levels of motor neuron proteins, which are necessary for the motor cells in the spine to function. Patients who receive gene therapy are more likely to survive, and they may have fewer permanent complications. The U.S. Food And Drug Administration has approved this treatment for use in children under two years old. However, it should be noted that this treatment has not yet been approved for adolescents and adults.