We have selected key publications on recent developments and important historical publications concerning phenylketonuria (PKU). Free summaries and slide sets are available to download. Reproduction of content owned by BioMarin on this page is authorised for non-commercial purposes. You may be required to clear additional rights if specific content depicts identifiable private individuals or includes third-party works. To use or reproduce content that is not owned by BioMarin (including but not limited to content compiled by BioMarin subject to a license from the original copyright holder), you may need to seek permission directly from the right-holders. Software or documents covered by industrial property rights, such as patents, trademarks, registered designs, logos, and names, are excluded from BioMarin’s reuse policy and are not licensed to you.
Publications
Burton et al. compared the long-term effectiveness of pegvaliase versus sapropterin and medical nutrition therapy (MNT), or MNT alone.
Publications
Harding et al. investigated longer-term overall outcomes and characterized individual participant responses to pegvaliase from the randomized trials gathered from PRISM-1, PRISM-2 and the open-label extension study 165-304.