Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial

Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial

Pegvaliase is a recombinant Anabaena variabilis phenylalanine ammonia lyase (PAL) enzyme under investigation for treatment of adult phenylketonuria (PKU). This manuscript describes results of a randomized discontinuation trial (RDT) designed to evaluate the effects of pegvaliase treatment on blood p...

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Bibliographic Details
Main Authors: Cary O. Harding, Roberto Amato, Mary Stuy, Nicola Longo, Barbara K. Burton, John Posner, Haoling H. Weng, Markus Meriläinen, Zhonghua Gu, Joy Jiang, Jerry Vockley
Format: Artigo
Language:English
Published: 2018
Online Access:https://doi.org/10.1016/j.ymgme.2018.03.003
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https://doi.org/10.1016/j.ymgme.2018.03.003

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