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

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-Alpan,1 Nicola Longo,2 Marie McDonald,3 Suma P Shankar,4,5 Raphael Schiffmann,6 Peter Chang,7 Yinghua Shen,7 Arian Pano7 1Lysosomal Disorders Unit, Fairfax, VA, 2University of Utah, Salt Lake City, UT, 3Department of Pediatrics, Duke University, Durham, NC, 4Department of Ophthalmology,...

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Bibliographic Details
Main Authors: Goker-Alpan O, Longo N, McDonald M, Shankar SP, Schiffmann R, Chang P, Shen Y, Pano A
Format: Article
Language:English
Published: Dove Medical Press 2016-05-01
Series:Drug Design, Development and Therapy
Subjects:
agalsidase alfa
efficacy
enzyme replacement therapy
Fabry disease
pediatric study
safety
Online Access:https://www.dovepress.com/an-open-label-clinical-trial-of-agalsidase-alfa-enzyme-replacement-the-peer-reviewed-article-DDDT

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https://www.dovepress.com/an-open-label-clinical-trial-of-agalsidase-alfa-enzyme-replacement-the-peer-reviewed-article-DDDT

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