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First 1.5 years of pegvaliase clinic: Experiences and outcomes
We present Boston Children's Hospital's clinic model for pegvaliase therapy in adults with phenylketonuria (PKU) and clinical outcomes in 46 patients over the first 1.5 years of commercial therapy. Approximately 70% (18/26) of patients starting pegvaliase achieved blood phenylalanine (Phe)...
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| Main Authors: | , , , , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Elsevier
2020-09-01
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| Serier: | Molecular Genetics and Metabolism Reports |
| Online adgang: | http://www.sciencedirect.com/science/article/pii/S2214426920300495 |
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