PYRUVATE DEHYDROGENASE COMPLEX DEFICIENCY DISEASE
Clinical trials for PYRUVATE DEHYDROGENASE COMPLEX DEFICIENCY DISEASE explained in plain language.
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New hope for rare metabolic disorder: drug aims to ease fatigue and improve lives
Disease control Recruiting nowThis study tests whether a medicine called glycerol phenylbutyrate (RAVICTI®) can help reduce fatigue and improve daily functioning in people with pyruvate dehydrogenase (PDH) deficiency, a rare genetic condition that affects energy production. About 15 children and adults aged 2…
Matched conditions: PYRUVATE DEHYDROGENASE COMPLEX DEFICIENCY DISEASE
Phase: PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Apr 26, 2026 20:02 UTC
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Scientists seek clues to rare metabolic disorder in new natural history study
Knowledge-focused Recruiting nowThis research study is for children and adults with pyruvate dehydrogenase complex deficiency (PDCD), a rare genetic disorder that affects energy production in cells. Researchers aim to learn more about the genetic causes, symptoms, and long-term outcomes by reviewing medical rec…
Matched conditions: PYRUVATE DEHYDROGENASE COMPLEX DEFICIENCY DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 17, 2026 05:47 UTC