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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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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2020
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7570269/ https://www.ncbi.nlm.nih.gov/pubmed/33123636 http://dx.doi.org/10.3390/ijns6030055 |