In a hopeful step for medicine, a 2-and-a-half-year-old child born with spinal muscular atrophy (SMA) has shown no symptoms of the genetic condition, all thanks to a gene-targeting drug that the mother took during late pregnancy.
A new study details this breakthrough case of prenatal therapy that holds promise for future treatment of the neurodegenerative disorder, which leads to the wasting of muscles over time and is usually diagnosed in infancy or early childhood.
