Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients <4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial

BACKGROUND: Sapropterin dihydrochloride, a synthetic formulation of BH(4), the cofactor for phenylalanine hydroxylase (PAH, EC 1.14.16.1), was initially approved in Europe only for patients ≥4 years with BH(4)-responsive phenylketonuria. The aim of the SPARK (Safety Paediatric efficAcy phaRmacokinet...

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Publicat a:Orphanet J Rare Dis
Autors principals: Muntau, Ania C., Burlina, Alberto, Eyskens, François, Freisinger, Peter, De Laet, Corinne, Leuzzi, Vincenzo, Rutsch, Frank, Sivri, H. Serap, Vijay, Suresh, Bal, Milva Orquidea, Gramer, Gwendolyn, Pazdírková, Renata, Cleary, Maureen, Lotz-Havla, Amelie S., Munafo, Alain, Mould, Diane R., Moreau-Stucker, Flavie, Rogoff, Daniela
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2017
Matèries:
Research
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5343543/
https://ncbi.nlm.nih.gov/pubmed/28274234
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0600-x
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