Dr Sean Smith | Fighting ALS at the Genetic Level: Real World Results for Tofersen

Amyotrophic lateral sclerosis (or ALS), also known as Motor Neuron Disease, is a devastating condition that gradually reduces a patient’s ability to move, speak, and ultimately breathe. Among the various forms of ALS, about 2% of cases are linked to a mutation in a gene called SOD1. For years, there has been no effective treatment for these patients. A new investigation conducted by Dr. Sean Smith and his colleagues at Washington University in St. Louis offers new hope for ALS patients.