SMA is a rare genetic disease that affects the central nervous system, voluntary skeletal muscle movement, and the peripheral nervous system (the connection between the central nervous system and the rest of the body). As a result, SMA affects basic motor functions such as walking, hand movements and feeding. After progression, the patients lose the ability to swallow, breathe and eventually die.
Zolgensma is a new one time gene therapy treatment for kids under the age of 2. It REPLACES the missing SMN1 gene with a new, working copy that helps motor neuron cells work properly and survive but it’s currently the most expensive drug in the world at $2.1 million. It is NOT a cure since it does not reverse damage already caused by SMA before treatment. However, with supportive therapy, it has by far shown the best results. It is recommended that the earlier the patient receives the treatment, the better.