Progress in Pediatric Cardiology
Volume 23, Issue 1 , Pages 39-48 , September 2007

Enzyme-deficiency metabolic cardiomyopathies and the role of enzyme replacement therapy

  • Priya S. Kishnani

      Affiliations

    • Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA
    • Corresponding Author InformationCorresponding author. Box 3528, Division of Medical Genetics, Duke University Medical Center, Durham, NC 27710, USA. Tel.: +1 919 684 2036; fax: +1 919 684 8944.
    • ,
  • Stephanie BurnsWechsler

      Affiliations

    • Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA
    • Division of Pediatric Cardiology, Duke University Medical Center, Durham, NC 27710, USA
    • ,
  • Jennifer S. Li

      Affiliations

    • Division of Pediatric Cardiology, Duke University Medical Center, Durham, NC 27710, USA

References 

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☆ PSK has received research grant support and honoraria from Genzyme Corporation. PSK is a member of the Pompe Disease Advisory Board for Genzyme Corporation. rhGAA, in the form of Genzyme's product, Myozyme™, has been approved by the US FDA and the European Union as therapy for Pompe disease. Duke University and inventors for the method of treatment and predecessors of the cell lines used to generate the enzyme (rhGAA) used in these clinical trials may benefit financially pursuant to the University's Policy on Inventions, Patents and Technology Transfer.

PII: S1058-9813(07)00006-9

doi: 10.1016/j.ppedcard.2007.05.005

Progress in Pediatric Cardiology
Volume 23, Issue 1 , Pages 39-48 , September 2007

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