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AMMONIA CONTROL AND NEUROCOGNITIVE OUTCOME AMONG UREA CYCLE DISORDER PATIENTS TREATED WITH GLYCEROL PHENYLBUTYRATE
BACKGROUND: Glycerol phenylbutyrate is under development for treatment of urea cycle disorders (UCDs), rare inherited metabolic disorders manifested by hyperammonemia and neurological impairment. METHODS: We report the results of a pivotal phase 3, randomized, double-blind, crossover trial comparing...
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Main Authors: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
2013
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Assuntos: | |
Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3557606/ https://ncbi.nlm.nih.gov/pubmed/22961727 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/hep.26058 |
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