Jerry Vockley, Md, Phd
Clinical trials sponsored by Jerry Vockley, Md, Phd, explained in plain language.
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Could a liver drug help kids and adults with rare metabolic disorder?
Disease control Recruiting nowThis study tests whether sodium phenylbutyrate (ACER-001), a drug already approved for another condition, can help people with MCAD deficiency caused by a specific gene mutation. About 24 participants aged 4 and older will take the drug and be monitored for safety and how well it…
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Jun 27, 2026 13:05 UTC
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Could a fatty acid drug stop dangerous sugar crashes in rare disease?
Disease control Recruiting nowThis study tests a drug called triheptanoin, already approved for similar conditions, to see if it can prevent dangerously low blood sugar in people with MCADD, a rare inherited disorder. About 24 participants aged 4 and older will take the medication and be monitored for safety …
Phase: PHASE2 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control
Last updated Jun 27, 2026 09:11 UTC