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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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Enzyme replacement therapy for Fabry disease: lessons from two alpha-galactosidase A orphan products and one FDA

Robert J Desnick1

  • 1Department of Human Genetics, Box 1498, Mount Sinai School of Medicine, Fifth Avenue at 100th St, New York, NY 10029, USA. rjdesnick@mssm.edu

Expert Opinion on Biological Therapy
|July 23, 2004
PubMed

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