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
Details:
- 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
