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Drug Design, Development and Therapy Volume 10, 2016 - Issue
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Original Research

An open-label clinical trial of agalsidase alfa enzyme replacement therapy in children with Fabry disease who are naïve to enzyme replacement therapy

Ozlem Goker-Alpan1 Lysosomal Disorders Unit, Fairfax, VA, USACorrespondence[email protected]
,
Nicola Longo2 University of Utah, Salt Lake City, UT, USA
,
Marie McDonald3 Department of Pediatrics, Duke University, Durham, NC, USA
,
Suma P Shankar4 Department of Ophthalmology, Emory University School of Medicine, Atlanta, GA, USA;5 Department of Human Genetics, Emory University School of Medicine, Atlanta, GA, USA
,
Raphael Schiffmann6 Institute of Metabolic Disease, Baylor Research Institute, Dallas, TX, USA
,
Peter Chang7 Shire, Lexington, MA, USA
,
Yinghua Shen7 Shire, Lexington, MA, USA
&
Arian Pano7 Shire, Lexington, MA, USA
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Pages 1771-1781 | Published online: 25 May 2016

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