Longevity & AgingsiRNA Drug Cemdisiran Slashes Hospitalizations in Myasthenia Gravis Trial
Cemdisiran, an investigational siRNA therapy that reduces complement component 5 (C5) production in the liver, dramatically cut hospitalizations and myasthenic crises in a phase III trial called NIMBLE. Compared to placebo, only 3.8% of cemdisiran-treated patients were hospitalized versus 15.3% on placebo. Total myasthenia gravis-related hospital days were just 1 in the treated group versus 92 in the placebo group over 24 weeks. The drug met its primary endpoint of improving daily-living scores, and rescue therapy use was also far lower in the treatment arm. Myasthenia gravis is a chronic autoimmune neuromuscular disease that worsens with age, making effective long-term management directly relevant to functional capacity and healthspan in older adults.
