Tofersen, SOD1 , and the Treatability of Amyotrophic Lateral Sclerosis

For years, amyotrophic lateral sclerosis (ALS) has universally been defined as a progressive, fatal disease with limited treatment options. Numerous therapeutic treatment trials failed to significantly slow disease progression or prolong survival. Until recently, there have been only 2 drugs approved in the US for treatment of ALS: riluzole and edaravone, which slow disease progression and prolong survival modestly. The reason for the lack of therapeutic success has been largely attributed to minimal understanding of the cause(s) of ALS and that treatment is generally initiated late in the pathological progression of disease, once neurodegeneration has become profound.