Demonstrating Success of a One-Dose Gene Therapy to Prevent Motor Neuronal Loss in Both Sporadic & Familial ALS

Time: 2:10 pm
day: Day Two Track A PM


  • Transitioning from monogenetic to sporadic populations: transformative progress to demonstrate efficacy in rodents
  • Outlining implications for ‘what’s next’, to drive AAV-UPF1 into clinical sporadic and familial ALS populations