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New drug may slow progression of ALS in small group of patients

By ABC News  
   December 23, 2025

A new drug may slow progression of — and even reverse — symptoms of a rare form of amyotrophic lateral sclerosis, or ALS, a new study published Monday finds. The drug, tofersen, targets a very specific mutation — SOD1 — which applies to only 2% of the ALS population. Among this group, the drug has the potential to slow muscle degeneration by targeting SOD1 mRNA, genetic material that tells the body how to make proteins, and reduces the proteins being made.

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