SPINRAZA targets an underlying cause of muscle weakness in SMA
People with spinal muscular atrophy (SMA) can’t make enough SMN protein, the protein their motor neurons need to function, because they have a mutated or deleted survival motor neuron 1 (SMN1) gene. The gene they do have, SMN2, does not produce enough protein for all of the body’s muscles. That’s where SPINRAZA can help.
SOURCE: Spinraza
