Hope of a Happy Life for Anna

Anna is an 18-year-old girl who suffers from an aggressive, fatal form of amyotrophic lateral sclerosis (ALS). In 2020, genetic testing revealed Anna’s shocking diagnosis of a debilitating nano-rare form of ALS, FUS P525L. Nobody expected ALS to exist within a 15-year-old girl, as it typically affects people between the ages of 40 and 70. Dr. Neil Shneider of the Eleanor and Lou Gehrig ALS Center at Columbia University in New York administered her Jacifusen, an ASO medicine he helped discover in partnership with Ionis Pharmaceuticals. He believes that Anna has had a remarkable response to the ASO and is optimistic about the treatment potentially leading her to a better life.