The Journal of Pediatrics
Volume 152, Issue 4 , Pages 563-570.e1 , April 2008

Safety and Efficacy of Enzyme Replacement Therapy with Agalsidase Beta: An International, Open-label Study in Pediatric Patients with Fabry Disease

  • J. Edmond Wraith, MB, ChB

      Affiliations

    • Royal Manchester Children’s Hospital, Manchester, United Kingdom
    • J. Edmond Wraith and Anna Tylki-Szymanska were equal contributors to this article.
    • ,
  • Anna Tylki-Szymanska, MD, PhD

      Affiliations

    • The Children’s Memorial Health Institute, Warsaw, Poland
    • Corresponding Author InformationReprint requests: A.Tylki-Szymanska, The Children’s Memorial Health Institute, Warsaw, Poland.
    • J. Edmond Wraith and Anna Tylki-Szymanska were equal contributors to this article.
    • ,
  • Nathalie Guffon, MD, PhD

      Affiliations

    • Hôpital Edouard Herriot, Lyon, France
    • ,
  • Y. Howard Lien, MD, PhD

      Affiliations

    • University of Arizona, Tucson, Arizona
    • ,
  • Michel Tsimaratos, MD, PhD

      Affiliations

    • Assistance Publique-Hôpitaux de Marseille la Timone-Enfants, Marseille, France
    • ,
  • Ashok Vellodi, MBBS

      Affiliations

    • Great Ormond Street Hospital for Children, London, United Kingdom
    • ,
  • Dominique P. Germain, MD, PhD

      Affiliations

    • University of Versailles-St Quentin en Yvelines and Centre de référence de la maladie de Fabry et des maladies héréditaires du tissu conjonctif, Assistance Publique-Hôpitaux de Paris, Paris, France

Received 28 December 2006 ,Revised 19 July 2007 ,Accepted 5 September 2007.

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 Sponsored by Genzyme Corporation.

 This trial has been registered at clinicaltrials.gov. The study ID# is NCT00074958.

 Conflict-of-Interest Disclosures: Dr Wraith has received honoraria and consulting fees from Genzyme; Dr Tylki-Szymanska has received honoraria and consulting fees from Genzyme; Dr Guffon received a research grant from Genzyme; Dr Lien has received research grants from Genzyme and Shire; Dr Tsimaratos has received honoraria from Genzyme and Shire; Dr Vellodi has received honoraria from Genzyme; and Dr Germain has receieved a research grant and consulting fees from Genzyme.

PII: S0022-3476(07)00852-9

doi: 10.1016/j.jpeds.2007.09.007

The Journal of Pediatrics
Volume 152, Issue 4 , Pages 563-570.e1 , April 2008

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