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AB015. Very early for pompe disease contribute to better outcomes: 7-year cohort study in Taiwan
BACKGROUND: Pompe disease is a lysosomal storage disorder characterized by the deficiency of acid α-glucosidase (GAA). Whether outcomes differ between very early (few days of age) and early (few weeks of age) ERT is unknown. In our series, 789,797 newborns were screened for Pompe disease. After 2010...
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| Veröffentlicht in: | Ann Transl Med |
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| 1. Verfasser: | |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
AME Publishing Company
2015
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4563481/ https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3978/j.issn.2305-5839.2015.AB015 |
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