Illuminating pharmacological treatments for spinal muscular atrophy

For spinal muscular atrophy (SMA) Awareness Month, we take a look at advances in the pharmacological treatment of this rare hereditary disease.

What is SMA?

SMA, a condition that progressively destroys motor neurons,1 affects approximately 1 in 10,000 live births and is the number one cause of infant death.2,3 Loss of signal between the motor neurons and the muscles leads to a gradual weakening of the muscles and eventual atrophy, affecting essential skeletal muscular activities such as walking, speaking, breathing, and swallowing.