Could a fatty acid drug tame a rare metabolic disease?
Disease control
Recruiting now
This early-stage study tests triheptanoin, a drug already approved for other conditions, in 6 children with pyruvate dehydrogenase complex (PDC) deficiency. The goal is to see if it can improve metabolic markers, reduce seizures, and lower hospital visits. The study focuses on sa…
Phase: PHASE1 • Sponsor: Jirair Krikor Bedoyan • Aim: Disease control
Last updated May 04, 2026 16:21 UTC