New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride

por Cary O Harding
Publicado 2010

A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials

por Barbara K. Burton, Anne Skalicky, Christoph Baerwald, Deborah A. Bilder, Cary O. Harding, Aaron B. Ilan, Elaina Jurecki, Nicola Longo, David T. Madden, H. Serap Sivri, Gisela Wilcox, Janet Thomas, Kathleen Delaney
Publicado 2021