317. Trifunctional protein deficiency
3 clinical trials,   8 drugs   (DrugBank: 4 drugs),   1 drug target gene,   1 drug target pathway

Searched query = "Trifunctional protein deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

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1 trial found
No.TrialIDDate_
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agemin
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PhaseCountries
1NCT01461304
(ClinicalTrials.gov)
November 30, 201125/10/2011Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy MetabolismDietary Therapy for Inherited Disorders of Energy MetabolismVery Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency;Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2);Mitochondrial Trifunctional Protein Deficiency;Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency;Glycogen Storage Disorders;Pyruvate Carboxylase Deficiency Disease;ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of;Barth SyndromeDrug: triheptanoinJerry Vockley, MD, PhDUltragenyx Pharmaceutical IncAvailable1 MonthN/AAllUnited States