•Presentations highlight clinical outcomes in pediatric PCQD subjects and translational evidence supporting tissue delivery and mitochondrial metabolic correction•Data support planned Phase 3 pivotal trial in PCQD following FDA Type C meeting
•Presentations highlight clinical outcomes in pediatric PCQD subjects and translational evidence supporting tissue delivery and mitochondrial metabolic correction•Data support planned Phase 3 pivotal trial in PCQD following FDA Type C meeting