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 disease in first human test

Disease control By invitation only

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 brain damage and seizures. The goal is to see if the drug is safe and can lower harmful acid levels in the body. R…

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

Phase 1 • Sponsor: Jerry Vockley, MD, PhD • Aim: Disease control

Last updated Jun 27, 2026 13:04 UTC

Combined D,L-2-hydroxyglutaric aciduria

Get alerted when new Combined D,L-2-hydroxyglutaric aciduria trials appear

Experimental drug targets rare brain disease in first human test