A benefit-risk analysis of pegvaliase for the treatment of phenylketonuria: A study of patients' preferences

ConclusionMost participants felt the burden of PKU on their daily lives, were dissatisfied with current treatments, and were willing to accept the risks of hypersensitivity reactions to achieve recommended blood Phe levels with pegvaliase treatment.
Source: Molecular Genetics and Metabolism Reports - Category: Genetics & Stem Cells Source Type: research