Spinraza

Spinraza is the only medical treatment that exists for SMA. The groundbreaking and revolutionary medicine stops the negative progress of the disease and additionally gives back some functions. The medicine is given by injection to the spine. The patient is given four doses during the first eight weeks, then a dose ever fourth month.

People with the most common form of SMA, lack the gene SMN1. This gene produces a vital protein that protects a type of nerve cells that sends signals to the bodies muscles. SMN1 has a “copy-gene”, SMN2. This gene doesn’t work completely. Spinraza helps SMN2, so that it can produce the protein that is normally produced by SMN1. Current studies show that babies that get the medicine right after birth, follow a normal development curve.