PYRUVATE DEHYDROGENASE COMPLEX DEFICIENCY DISEASE
Clinical trials for PYRUVATE DEHYDROGENASE COMPLEX DEFICIENCY DISEASE explained in plain language.
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Hope for rare metabolic disorder: new drug targets fatigue in PDH deficiency
Disease control Recruiting nowThis study tests whether a drug called glycerol phenylbutyrate (RAVICTI®) can reduce fatigue and improve daily life for people with pyruvate dehydrogenase (PDH) deficiency, a rare genetic condition that affects energy production. About 15 children and young adults (ages 2 to 25) …
Matched conditions: PYRUVATE DEHYDROGENASE COMPLEX DEFICIENCY DISEASE
Phase: PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 22, 2026 12:03 UTC
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Researchers launch study to unravel rare metabolic disorder
Knowledge-focused Recruiting nowThis study is observing children and adults with pyruvate dehydrogenase complex deficiency (PDCD), a rare genetic disorder that affects energy production in cells. Researchers will collect medical history, questionnaire responses, and genetic data from 150 participants to better …
Matched conditions: PYRUVATE DEHYDROGENASE COMPLEX DEFICIENCY DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Jun 22, 2026 12:00 UTC