Signs and Symptoms

Signs and Symptoms

One of the pathological hallmarks of spinal muscular atrophy (SMA) is the degeneration of anterior horn cells in the spinal cord and motor nuclei in the lower brainstem.1 Alpha motor neurons are preferentially affected, so the disease is limited to the motor system...
Prognosis

Prognosis

The disease course and prognosis of spinal muscular atrophy (SMA) depends heavily on the clinical classification in a particular patient. For example, the prognosis of children with SMA type 0 is very poor while the prognosis in SMA type 4 patients is relatively good....
Disease-Modifying Therapies for Treating SMA

Disease-Modifying Therapies for Treating SMA

Until the approval of nusinersen (Spinraza), treatment for spinal muscular atrophy was largely supportive. While patients with SMA enjoy benefits from respiratory therapy, physical therapy, nutrition support, etc., these therapies are aimed at maximizing residual...