Historical Perspective on Clinical Trials of Carnitine in Children and Adults
| Autor: | Neil R. M. Buist |
|---|---|
| Rok vydání: | 2016 |
| Předmět: |
Adult
medicine.medical_specialty Orphan Drug Production Nutritional Sciences Medicine (miscellaneous) Physiology Child Nutrition Sciences Biology History 21st Century Lipid Metabolism Inborn Errors law.invention Food and drug administration 03 medical and health sciences 0302 clinical medicine Muscular Diseases Randomized controlled trial law Carnitine 030225 pediatrics Internal medicine medicine Humans Hyperammonemia Child Clinical Trials as Topic Nutrition and Dietetics Ethical issues Infant Lipid metabolism History 20th Century medicine.disease Clinical trial Malnutrition Endocrinology Carnitine Acyltransferases Dietary Supplements Administration Intravenous Cardiomyopathies Deficiency Diseases Energy Metabolism Primary Carnitine Deficiency Metabolism Inborn Errors 030217 neurology & neurosurgery medicine.drug |
| Zdroj: | Annals of Nutrition and Metabolism. 68:1-4 |
| ISSN: | 1421-9697 0250-6807 |
| Popis: | The metabolic roles of carnitine have been greatly clarified over the past 50 years, and it is now well established that carnitine is a key player in mitochondrial generation of energy and metabolism of acetyl coenzyme A. A therapeutic role for carnitine in treatment of nutritional deficiencies in infants and children was first demonstrated in 1958, and since that time it has been used to treat a number of inborn errors of metabolism. Carnitine was approved by the US Food and Drug Administration in 1985 for treatment of ‘primary carnitine deficiency', and later in 1992 for treatment of ‘secondary carnitine deficiency', a definition that included the majority of relevant metabolic disorders associated with low or abnormal plasma carnitine levels. Today, carnitine treatment of inborn errors of metabolism is a safe and integral part of many treatment protocols, and a growing interest in carnitine has resulted in greater recognition of many causes of carnitine depletion. Notwithstanding, there is still a lack of data from randomized clinical trials, even on the use of carnitine in inborn errors of metabolism, although ethical issues may be a contributing factor in this regard. |
| Databáze: | OpenAIRE |
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