Pharmacokinetic, pharmacodynamic, and immunogenic rationale for optimal dosing of pegvaliase, a PEGylated bacterial enzyme, in adult patients with phenylketonuria-Reference-Cited by-同舟云学术

Pharmacokinetic, pharmacodynamic, and immunogenic rationale for optimal dosing of pegvaliase, a PEGylated bacterial enzyme, in adult patients with phenylketonuria

Published:2021-05-31 Issue:5 Volume:14 Page:1894-1905
ISSN:1752-8054
Container-title:Clinical and Translational Science
language:en
Short-container-title:Clinical Translational Sci

Author:

Qi Yulan¹,Patel Gina¹,Henshaw Joshua¹,Gupta Soumi¹,Olbertz Joy¹,Larimore Kevin¹,Harding Cary O.²,Merilainen Markus¹,Zori Roberto³,Longo Nicola⁴,Burton Barbara K.⁵,Li Mingjin¹,Gu Zhonghua¹,Zoog Stephen J.¹,Weng Haoling H.¹,Schweighardt Becky¹

Affiliation:

1. BioMarin Pharmaceutical Inc Novato CA USA

2. Department of Molecular and Medical Genetics Oregon Health & Science University Portland OR USA

3. Division of Genetics and Metabolism University of Florida Gainesville FL USA

4. Division of Medical Genetics University of Utah Salt Lake City UT USA

5. Division of Genetics, Birth Defects and Metabolism Ann & Robert H. Lurie Children's Hospital Chicago and Northwestern University Feinberg School of Medicine Chicago IL USA

Funder

BioMarin Pharmaceutical

Publisher

Wiley

Subject

General Pharmacology, Toxicology and Pharmaceutics,General Biochemistry, Genetics and Molecular Biology,General Medicine,General Neuroscience

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/cts.13043

Reference28 articles.

1. The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6R-tetrahydrobiopterin): a phase II, multicentre, open-label, screening study

2. Systematic Review and Meta-Analysis of Neuropsychiatric Symptoms and Executive Functioning in Adults With Phenylketonuria

3. High phenylalanine levels directly affect mood and sustained attention in adults with phenylketonuria: a randomised, double-blind, placebo-controlled, crossover trial