Uploaded April 2023 | Updated September 2026, 2 weeks ago
A new drug has been approved by the Food and Drug Administration (FDA) for a rare, inherited form of amyotrophic lateral sclerosis (ALS) caused by mutations in the gene SOD1. Called tofersen, the drug — developed by Biogen Inc. and based in part on research conducted at Washington University School of Medicine in St. Louis — slowed progression of the fatal, paralyzing disease. Some participants in the phase 3 clinical trial experienced a stabilization of muscle strength and control, encouraging signs for a disease that is normally characterized by continual decline.
For more information: https://medicine.wustl.edu/news/drug-for-rare-form-of-als-based-in-part-on-washu-research-approved-by-fda/
Video by Huy Mach and Tamara Bhandari/Washington University School of Medicine in St. Louis
A new drug has been approved by the Food and Drug Administration (FDA) for a rare, inherited form of amyotrophic lateral sclerosis (ALS) caused by mutations in the gene SOD1. Called tofersen, the drug — developed by Biogen Inc. and based in part on research conducted at Washington University School of Medicine in St. Louis — slowed progression of the fatal, paralyzing disease. Some participants in the phase 3 clinical trial experienced a stabilization of muscle strength and control, encouraging signs for a disease that is normally characterized by continual decline.
For more information: https://medicine.wustl.edu/news/drug-for-rare-form-of-als-based-in-part-on-washu-research-approved-by-fda/
Video by Huy Mach and Tamara Bhandari/Washington University School of Medicine in St. Louis
