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Story perspectives

Tofersen Breakthrough: Promising ALS Treatment Reduces Mortality Risk

12/22/2025

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Story summary
  • Tofersen shows promise in slowing progression of SOD1-ALS, a rare form affecting about 2% of ALS patients.
  • A recent study shows over 20% of participants gained strength and function after three years.
  • The spinal-injection treatment yields a 48% reduction in mortality risk for early users.
  • Jessica Morris reports meaningful quality-of-life improvements and some regained mobility.
  • Researchers say tofersen could advance treatment for other ALS forms.