New hope for rare disorder: testing drug to prevent dangerous blood sugar drops
Disease control
Recruiting now
This study is testing if a medication called triheptanoin is safe and can help prevent low blood sugar (hypoglycemia) in people aged 4 and older with Medium-chain Acyl-CoA Dehydrogenase Deficiency (MCADD), a rare inherited metabolic disorder. Researchers will monitor participants…
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC