MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Clinical trials for MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY explained in plain language.
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Trial tests if existing drug can help rare metabolic disorder
Disease control TerminatedThis study is testing if a medication called sodium phenylbutyrate (ACER-001), already approved for a different condition, can help people aged 10 and older with a specific form of MCAD deficiency. Researchers want to see if taking the drug for 4 weeks allows patients to fast lon…
Matched conditions: MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Apr 04, 2026 04:51 UTC
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Hope for rare disorder: drug trial aims to stop dangerous blood sugar crashes
Disease control TerminatedThis study is testing if a medication called triheptanoin can safely prevent low blood sugar (hypoglycemia) in adults with a rare inherited condition called MCADD. Eight participants will take the drug for about 9 weeks while researchers monitor for side effects and check if it h…
Matched conditions: MEDIUM-CHAIN ACYL-COA DEHYDROGENASE DEFICIENCY
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Mar 25, 2026 14:08 UTC