Guide To Spinal Muscular Atrophy Types And Treatment

Gene Therapy

Spinal muscular atrophy patients need gene therapy as part of their treatment. They receive treatment that replaces the defective or missing SMN1 gene. This is a one-time treatment! After gene therapy, patients have a working copy of the gene. It restores some of the motor function, especially in their spine!

Individuals with this condition who undergo gene therapy have a higher survival rate. They also have fewer permanent complications! However, the United States Food and Drug Administration has only approved this treatment for children under two years old. Older children and adults with this condition are not currently included in this!