Triheptanoin treatment in patients with pediatric cardiomyopathy associated with long chain-fatty acid oxidation disorders

Author:

Vockley J.,Charrow J.,Ganesh J.,Eswara M.,Diaz G.A.,McCracken E.,Conway R.,Enns G.M.,Starr J.,Wang R.,Abdenur J.E.,Sanchez-de-Toledo J.,Marsden D.L.

Funder

NIH

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference30 articles.

1. Management and outcome in 75 individuals with long-chain fatty acid oxidation defects: results from a workshop;Spiekerkoetter;J.Inherit.Metab Dis.,2009

2. M. Lindner, G. Gramer, G. Haege, J. Fang-Hoffmann, K.O. Schwab, U. Tacke, F.K. Trefz, E. Mengel, U. Wendel, M. Leichsenring, P. Burgard, G.F. Hoffmann, Efficacy and outcome of expanded newborn screening for metabolic diseases–report of 10years from South-West Germany Orphanet J Rare Dis 6 (2011) 44.

3. Clinical and biological features at diagnosis in mitochondrial fatty acid beta-oxidation defects: a French pediatric study of 187 patients;Baruteau;J Inherit.Metab Dis.,2012

4. Inherited cardiomyopathies;Kelly;N. Engl. J. Med.,1994

5. Long-Term Major Clinical Outcomes in Patients with Long Chain Fatty Acid Oxidation Disorders before and after Transition to Triheptanoin Treatment—A Retrospective Chart Review Molecular Genetics and Metabolism;Vockley,2015

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