Publications
PKU.expert single slide summaries
14 papers were highlighted in our literature highlights. The first paper by Harding et al. demonstrated that long-term pegvaliase substantially and durably decreased blood Phe levels, with 71.3% achieving a clinically significant blood Phe reduction of ≤600 µmol/L. Burton et al. showed that pegvaliase induced a sustained reduction in target blood Phe concentrations compared with medical nutrition therapy, with or without sapropterin. Rocha et al. highlighted that there is an unmet treatment need due to the limitations and burden of long-term reliance on medical nutrition therapy.