International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria.

Muntau, Ania C; Adams, Darius J; Bélanger-Quintana, Amaya; Bushueva, Tatiana V; Cerone, Roberto; Chien, Yin-Hsiu; Chiesa, Ana; Coskun, Turgay; de Las Heras, Javier; Feillet, François; Katz, Rachel; Lagler, Florian; Piazzon, Flavia; Rohr, Fran; van Spronsen, Francjan J; Vargas, Paula; Wilcox, Gisela; Bhattacharya, Kaustuv.
Mol Genet Metab; 127(1): 1-11, 2019 05.
Artigo em Inglês | MEDLINE | ID: mdl-31103398