FDA OKs First-of-its-Kind Treatment for Rare Form of ALS.

The article reports on the Qalsody, a treatment for rare form of amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS) ), that has been approved by the U.S. Food and Drug Administration (FDA). It informs the controversy surrounding the list price for Qalsody and provides an overview of the randomized, double-blind, placebo-controlled clinical on the safety and effectiveness of Qalsody.