A Deficiency of Carnitine–Acylcarnitine Translocase in the Inner Mitochondrial Membrane

Charles A. Stanley, Daniel Hale, Gerard T. Berry, Susan Deleeuw, Jay Boxer, Jean Paul Bonnefont

Research output: Contribution to journalArticlepeer-review

161 Scopus citations

Abstract

MITOCHONDRIAL oxidation of fatty acids provides the chief source of energy during prolonged fasting as well as for cardiac muscle and skeletal muscle during exercise. In the past decade, 10 genetic defects of this pathway have been recognized in infants and children.1 2 3 4 5 6 7 Although each of these disorders is rare, failure to recognize them can deprive patients of lifesaving therapy. Patients with these defects present with coma after a period of starvation and have hypoketosis — that is, their serum ketone concentrations are low. They may also have cardiomyopathy and muscle weakness. Three of the disorders affect the carnitine cycle, illustrated…

Original languageEnglish (US)
Pages (from-to)19-23
Number of pages5
JournalNew England Journal of Medicine
Volume327
Issue number1
DOIs
StatePublished - Jul 2 1992

All Science Journal Classification (ASJC) codes

  • General Medicine

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