Drug for rare form of ALS benefits some patients

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: medicine.wustl...
Video by Huy Mach and Tamara Bhandari/Washington University School of Medicine in St. Louis

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SOD1 gene

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Tofersen

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