There is a paradigm shift in the way clinicians think about and provide treatment — a shift toward preventative, personalized, and proactive medicine, especially for genetic conditions such as spinal muscular atrophy (SMA).
The rare, neuromuscular, inherited condition affects 1 in every 6000 to 10,000 children. In the United States, an estimated 10,000 to 25,000 children and adults have a SMA diagnosis. Due to the genetic nature of the disorder, SMA is always present at birth; however, it may take longer for symptoms to appear, depending on the disease type. In SMA, muscle weakness, fasciculations, and atrophy are due to the loss of motor neurons disrupting nerve signaling to skeletal muscles.