Key result
Bezafibrate shows no benefit for patients with CPTII and VLCAD deficiencies.
Bezafibrate shows no benefit in CPTII/VLCAD deficiency; should not change practice and leaves open need for alternative therapies.
Journal of Inherited Metabolic DiseaseVolume 38, Issue 2 p. 373-374 Letter to the Editor No effect of bezafibrate in patients with CPTII and VLCAD deficiencies Mette Cathrine Ørngreen, Corresponding Author Mette Cathrine Ørngreen [email protected] Neuromuscular Clinic and Research Unit, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark [email protected]Search for more papers by this authorJohn Vissing, John Vissing Neuromuscular Clinic and Research Unit, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, DenmarkSearch for more papers by this authorPascal Laforét, Pascal Laforét Centre de Référence de Pathologie Neuromusculaire Paris-Est, Groupe Hospitalier Pitié-Salpêtrière, Assistance Publique-Hôpitaux de Paris, Paris, FranceSearch for more papers by this author Mette Cathrine Ørngreen, Corresponding Author Mette Cathrine Ørngreen [email protected] Neuromuscular Clinic and Research Unit, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark [email protected]Search for more papers by this authorJohn Vissing, John Vissing Neuromuscular Clinic and Research Unit, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, DenmarkSearch for more papers by this authorPascal Laforét, Pascal Laforét Centre de Référence de Pathologie Neuromusculaire Paris-Est, Groupe Hospitalier Pitié-Salpêtrière, Assistance Publique-Hôpitaux de Paris, Paris, FranceSearch for more papers by this author First published: 21 October 2014 https://doi.org/10.1007/s10545-014-9779-3Citations: 22 Communicated by: Verena Peters Read the full textAboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onEmailFacebookTwitterLinkedInRedditWechat No abstract is available for this article. References Astrand PO, Rodahl K, Dahl HA, Stromme SB (2003) Textbook of work physiology. Physiological bases of exercise, Champaign: Human Kinetics. Bonnefont JP, Bastin J, Behin A, Djouadi F (2009) Bezafibrate for an inborn mitochondrial beta-oxidation defect. N Engl J Med, 360 (8): 838–840, . Djouadi F, Bastin J (2008) PPARs as therapeutic targets for correction of inborn mitochondrial fatty acid oxidation disorders. J Inherit Metab Dis, 31 (2): 217–225, . Orngreen MC, Olsen DB, Vissing J (2002) Exercise tolerance in carnitine palmitoyltransferase II deficiency with IV and oral glucose. Neurology, 59 (7): 1046–1051, . Orngreen MC, Ejstrup R, Vissing J (2003) Effect of diet on exercise tolerance in carnitine palmitoyltransferase II deficiency. Neurology, 61 (4): 559–561, . Orngreen MC, Madsen KL, Preisler N, Andersen G, Vissing J, Laforet P (2014) Bezafibrate in skeletal muscle fatty acid oxidation disorders: a randomized clinical trial. Neurology, 82 (7): 607–613, 3963421 Citing Literature Volume38, Issue2March 2015Pages 373-374 ReferencesRelatedInformation
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Ørngreen et al. (2014) conducted a letter in CPTII and VLCAD deficiencies. Bezafibrate was evaluated. Bezafibrate had no effect in patients with CPTII and VLCAD deficiencies.
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