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

Authors

Susan A. Berry, University of Minnesota
Uta Lichter-Konecki, George Washington University
George A. Diaz, Icahn School of Medicine at Mount Sinai
Shawn E. McCandless, Case Western Reserve University
William Rhead, The Medical College of Wisconsin
Wendy Smith, Maine Medical Center, Portland, MEFollow
Cynthia Lemons, National Urea Cycle Disorders Foundation, Pasadena, CA
Sandesh Nagamani, Baylor College of Medicine
Dion Coakley, Hyperion Therapeutics, San Francisco, CA
Masoud Mokhtarani, San Francisco, CA
Bruce F. Scharschmidt, Hyperion Therapeutics, San Francisco, CA
Brendan Lee, Baylor College of MedicineFollow

Document Type

Journal Article

Publication Date

5-2014

Journal

Molecular Genetics and Metabolism

Volume

Volume 112, Issue 1

Inclusive Pages

17-24

Keywords

Phenylbutyrates--adminstration & dosage; Phenylbutyrates--adverse effects; Urea Cycle Disorders, Inborn--drug therapy

APA Citation

Berry, S.A., Lichter-Konecki, U., Diaz, G.A., McCandless, S.E., Rhead, W. et al. (2014). Glycerol phenylbutyrate treatment in children with urea cycle disorders: pooled analysis of short and long-term ammonia control and outcomes. Molecular Genetics and Metabolism, 112(1), 17-24.

Peer Reviewed

1