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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| Autores principales: | , , , , , , , , , , |
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| Formato: | Artigo |
| Lenguaje: | inglés |
| Publicado: |
2018
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| Acceso en línea: | https://doi.org/10.1016/j.ymgme.2018.03.003 |
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