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The Kuvan(®) Adult Maternal Paediatric European Registry (KAMPER) Multinational Observational Study: Baseline and 1-Year Data in Phenylketonuria Patients Responsive to Sapropterin
Introduction: Sapropterin dihydrochloride (Kuvan(®)), a synthetic 6R-diastereoisomer of tetrahydrobiopterin (BH(4))(,) is approved in Europe for the treatment of patients aged ≥4 years with hyperphenylalaninaemia (HPA) due to BH(4)-responsive phenylalanine hydroxylase (PAH) deficiency, in conjunctio...
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| Publicat a: | JIMD Rep |
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| Autors principals: | , , , , , , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
Springer Berlin Heidelberg
2015
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4484899/ https://ncbi.nlm.nih.gov/pubmed/25822821 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2015_425 |
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