Translation of Gene Therapy Brain Biomarkers from Nonclinical Science to Clinical Trials: A Case Study in Canavan Disease

Time: 2:30 pm
day: Clinical Stream Day 1 PM

Details:

  • MRI and MRS evaluations of candidate biomarkers 
  • Preclinical assessments in a murine model
  • Clinical evidence by magnetic resonance spectroscopy in humans by standardized spectrum analyses
  • Potential uses in clinical trials to support regulatory activities

Speakers: