MEDIUM CHAIN ACYL COA DEHYDROGENASE DEFICIENCY
Clinical trials for MEDIUM CHAIN ACYL COA DEHYDROGENASE DEFICIENCY explained in plain language.
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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
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Experimental drug aims to help rare metabolic disorder patients handle fasting
Disease control TerminatedThis study is testing if a medication called sodium phenylbutyrate can help people with a rare genetic disorder called MCAD deficiency. The main goal is to see if the drug allows patients to safely fast for longer periods without developing low blood sugar. The trial involves 24 …
Matched conditions: MEDIUM CHAIN ACYL COA DEHYDROGENASE DEFICIENCY
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Mar 12, 2026 13:51 UTC