Until recently, doctors were only able to treat the symptoms of neuromuscular conditions. New, exciting innovations have meant that pharmacological treatments now exist for certain neuromuscular conditions.
Spinraza, which is the first drug approved by the FDA to treat spinal muscular atrophy (SMA). Unfortunately, SMA is often lethal within two years of birth, but by increasing the amount of a certain protein, Spinraza has shown quite promising results in patients, including at Stanford Children’s Health.
For example, here is Zoe’s remarkable success story.
Lumizyme, which is a drug that helps treat pompe disease, a genetic condition that results in an enzyme deficiency that causes cells in the muscles to break down. This enzyme is essential for breaking down a kind of sugar that is found in muscle cells. Lumizyme works to replace that enzyme so that the cells can remain healthy.