Long Chain Fatty Acid Oxidation Disorders
Conditions
Keywords
Expanded Access, Compassionate Use, Carnitine Palmitoyltransferase Deficiency, CPT I, CPT II, Very Long Chain acyl-CoA Dehydrogenase Deficiency, VLCAD, Long Chain 3-hydroxy-acyl-CoA Dehydrogenase Deficiency, LHCAD, Trifunctional Protein Deficiency, TFP, Carnitine-acylcarnitine Translocase Deficiency, CACT, LC-FAOD
Brief summary
Expanded access may be provided for qualified patients who have limited treatment options and are not eligible for a clinical trial.
Detailed description
Long Chain Fatty Acid Oxidation Disorders (LC-FAOD) and other Non-FAOD conditions, where applicable: Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies. For full details, please visit the links provided below.
Interventions
DRUGTriheptanoin
Liquid for oral (PO) or enteral tube administration
Sponsors
Ultragenyx Pharmaceutical Inc
Eligibility
Sex/Gender
ALL
Inclusion criteria
\- The participant must not be eligible for a UX007 clinical trial
Contacts
CONTACTEarly Access
STUDY_DIRECTORMedical Director
Ultragenyx Pharmaceutical Inc
Outcome results
None listed