COMBINED D,L-2-HYDROXYGLUTARIC ACIDURIA

Clinical trials for COMBINED D,L-2-HYDROXYGLUTARIC ACIDURIA explained in plain language.

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Experimental drug targets rare brain acid disorder in first human test

Disease control ENROLLING_BY_INVITATION

This study tests a drug called ACER-001 (sodium phenylbutyrate) in a single patient with combined D,L-2-hydroxyglutaric aciduria, a rare genetic disorder that causes harmful acid buildup in the body. The goal is to see if the drug is safe and can reduce acid levels, seizures, and…

Matched conditions: COMBINED D,L-2-HYDROXYGLUTARIC ACIDURIA

Phase: PHASE1 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control

Last updated Jun 25, 2026 00:23 UTC

COMBINED D,L-2-HYDROXYGLUTARIC ACIDURIA

Get alerted when new COMBINED D,L-2-HYDROXYGLUTARIC ACIDURIA trials appear

Experimental drug targets rare brain acid disorder in first human test