New ALS drug stabilizes decline with a trend toward improved strength, mobility for some

Historically, people with amyotrophic lateral sclerosis (ALS) experience a relentless decline of neurological function that eventually robs them of the ability to move, speak, eat or breathe. Now, researchers from Washington University School of Medicine in St. Louis and collaborators report that long-term use of tofersen, a new drug approved by the Food and Drug Administration (FDA) for a genetic form of this deadly illness, delays symptom progression and death and in about one-quarter of participants leads to stabilization or improvement.