Spinal Muscular Atrophy (SMA) is rare, progressive neuromuscular disease and a leading genetic cause of infant death when left untreated. SMA causes irreversible loss of motor neurons, which can rob infants of their ability to walk, swallow and even breathe. If left untreated in its most severe forms, 90% of children require permanent feeding and breathing support or pass away by their second birthday. SMA can progress quickly, making early diagnosis and treatment crucial.
Zolgensma is the only SMA treatment designed to directly address the genetic root cause of the disease by replacing the function of the missing or non-working SMN1 gene with a single, one-time dose.
Currently, more than 40 states screen newborns for SMA.
Learn more here.
