Effects of triheptanoin (UX007) in patients with long‐chain fatty acid oxidation disorders: Results from an open‐label, long‐term extension study

Long‐chain fatty acid oxidation disorders (LC‐FAOD) are autosomal recessive conditions that impair conversion of long‐chain fatty acids into energy, leading to significant clinical symptoms. Triheptanoin is a highly purified, 7‐carbon chain triglyceride approved in the United States as a source of c...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Veröffentlicht in:	Journal of inherited metabolic disease 2021-01, Vol.44 (1), p.253-263
Hauptverfasser:	Vockley, Jerry, Burton, Barbara, Berry, Gerard, Longo, Nicola, Phillips, John, Sanchez‐Valle, Amarilis, Chapman, Kimberly, Tanpaiboon, Pranoot, Grunewald, Stephanie, Murphy, Elaine, Lu, Xiaoxiao, Cataldo, Jason
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Adverse events Calories Cardiomyopathies - metabolism Cardiomyopathy Child Child, Preschool Clinical trials Diarrhea Fatty acids Fatty Acids - metabolism Female Humans Hypoglycemia Hypoglycemia - metabolism Infant Lipid Metabolism, Inborn Errors - drug therapy Lipid Metabolism, Inborn Errors - metabolism long‐chain fatty acid oxidation disorders (LC‐FAOD) Male Middle Aged Original Oxidation Oxidation-Reduction - drug effects Patients Rhabdomyolysis Rhabdomyolysis - metabolism Triglycerides - administration & dosage triheptanoin (UX007) United Kingdom United States Vomiting Young Adult
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Schreiben Sie den ersten Kommentar!