AVXS-401 / Novartis - LARVOL DELTA

Efficacy and Safety of CSF- Delivered AVXS-401 in Mice and Non-Human Primates for the Treatment of Friedreich’s Ataxia (ASGCT 2020) - "Dose escalation studies by intrathecal (IT) administration in NHP show a dose correlation between mice and NHP by ddPCR quantification of vector genomes. Together these preclinical data show that AVXS-401 is suitable for first-in-human studies."

MRI • Myoglobin