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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MDPI AG,
2020-07-01T00:00:00Z.
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