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Recent Developments, Utilization, and Spending Trends for Pompe Disease Therapies
BACKGROUND: Pompe disease is a rare condition, with an incidence rate estimated to be between 1 in 40,000 and 1 in 300,000 live births worldwide. For an infant who contracts the disease, which is an inherited metabolic myopathy caused by deficiency of the acid alpha-glucosidase (GAA) enzyme in lysos...
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| Hlavní autoři: | , , |
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| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
Engage Healthcare Communications, LLC
2012
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| Témata: | |
| On-line přístup: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4046468/ https://ncbi.nlm.nih.gov/pubmed/24991319 |
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