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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Pubblicato in:JIMD Rep
Autori principali: Trefz, Friedrich K., Muntau, Ania C., Lagler, Florian B., Moreau, Flavie, Alm, Jan, Burlina, Alberto, Rutsch, Frank, Bélanger-Quintana, Amaya, Feillet, François
Natura: Artigo
Lingua:Inglês
Pubblicazione: Springer Berlin Heidelberg 2015
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Article
Accesso online: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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