Nutritional status of adults with phenylketonuria on pegvaliase: A 15-month prospective study
Author:
Funder
BioMarin Pharmaceutical
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology
Reference33 articles.
1. Phenylalanine hydroxylase deficiency: diagnosis and management guideline;Vockley;Genet. Med.,2014
2. Pegvaliase for the treatment of phenylketonuria: results of a long-term phase 3 clinical trial program (PRISM);Thomas;Mol. Genet. Metab.,2018
3. Adherence to clinic recommendations among patients with phenylketonuria in the United States;Jurecki;Mol. Genet. Metab.,2017
4. Natural history of children and adults with phenylketonuria in the NBS-PKU connect registry;Kenneson;Mol. Genet. Metab.,2021
5. A benefit-risk analysis of pegvaliase for the treatment of phenylketonuria: a study of patients’ preferences;SriBhashyam;Mol Genet Metab Rep,2019
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