An experimental drug could help young people with a rare form of ALS, researchers at New York’s Columbia University Irving Medical Center announced on Thursday.
Known as “ulefnersen,” the therapy showed promise in treating patients with FUS-ALS, which is caused by a genetic mutation in a gene called FUS. The gene provides instructions for making a protein and plays a crucial role in cellular processes. While the mutations are only responsible for between one and two percent of ALS cases, they cause some of the most aggressive forms of ALS that begin in adolescents and young adults.
