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...

Descripción completa

Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5343543/
https://www.ncbi.nlm.nih.gov/pubmed/28274234
http://dx.doi.org/10.1186/s13023-017-0600-x

Ejemplares similares

Ejemplares similares