The drug, tofersen, was developed specifically for patients with SOD1-ALS, a rare form of the disease that occurs because of mutation in the SOD1 gene.
A new drug has been approved by the Food and Drug Administration (FDA) for a rare, inherited form of amyotrophic lateral sclerosis (ALS). Called tofersen, the drug developed by Biogen Inc. and based in part on research conducted at Washington University School of Medicine in St. Louis slows the progression of the deadly, paralyzing disease.
FDA granted accelerated approval of QALSODY based on a reduction of neurofilament, a marker of neurodegeneration1Superoxide dismutase 1 -amyotrophic lateral sclerosis is a devastating, uniformly. | April 25, 2023