Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

Amyotrophic lateral sclerosis (ALS) is a progressive disease of the nervous system. It affects nerve cells in the brain and spinal cord called motor neurons. Motor neurons control muscle movement and ...

The new data show that an axonal injury and neurodegeneration biomarker was reduced in nearly all study participants treated with Spinraza. The evaluates clinical outcomes and safety following ...