Spinal Muscular Atrophy (SMA) is a rare and devastating genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, death.
Years ago, doctors who diagnosed patients with SMA told parents to take their child home and make them comfortable and love them, because it wasn’t likely that the child would live to reach a milestone like their second birthday.
Now, with advances in treatment, families and caregivers who have a child with SMA have multiple options. Earlier this year, the FDA approved a gene therapy called Zolgensma that addresses the genetic root cause of the disease. . Zolgensma is approved to treat pediatric patients less than 2 years of age with SMA.
Learn more here.