Establishing Pompe Disease Newborn Screening: The Role of Industry

When clinical trials for enzyme replacement therapy for Pompe disease commenced, a need for newborn screening (NBS) for Pompe disease was recognized. Two methods for NBS for Pompe disease by measuring acid α-glucosidase in dried blood spots on filter paper were developed in an international collabor...

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Bibliographic Details
Main Author: Joan M. Keutzer (Author)
Format: Book
Published: MDPI AG, 2020-07-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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