Glycerol phenylbutyrate treatment in children with urea cycle disorders: Pooled analysis of short and long-term ammonia control and outcomes

Susan A. Berry(University of Minnesota), Brendan Lee(Baylor College of Medicine), George A. Díaz(Icahn School of Medicine at Mount Sinai), Dion F. Coakley, Wendy E. Smith(Maine Medical Center), Bruce F. Scharschmidt, Cynthia LeMons(National Urea Cycle Disorders Foundation), Masoud Mokhtarani, Uta Lichter‐Konecki(Children's National), Shawn E. McCandless(University Hospitals of Cleveland), Sandesh C.S. Nagamani(Baylor College of Medicine), William J. Rhead(Children's Hospital of Wisconsin)
Molecular Genetics and Metabolism
February 22, 2014
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