Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2 months to 2 years-Reference-Cited by-同舟云学术

Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2 months to 2 years

Published:2017-11 Issue:3 Volume:122 Page:46-53
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Berry Susan A.,Longo Nicola^ORCID,Diaz George A.,McCandless Shawn E.,Smith Wendy E.,Harding Cary O.,Zori Roberto^ORCID,Ficicioglu Can,Lichter-Konecki Uta,Robinson Beth,Vockley Jerry

Funder

Oregon Clinical and Translational Research Institute

National Center for Advancing Translational Sciences

National Institutes of Health

NIH Roadmap for Medical Research

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference25 articles.

1. Urea Cycle Disorders Overview;Mew,1993

2. Neonatal mortality and outcome at the end of the first year of life in early onset urea cycle disorders—review and meta-analysis of observational studies published over more than 35 years;Burgard;J. Inherit. Metab. Dis.,2016

3. Clinical outcomes of neonatal onset proximal versus distal urea cycle disorders do not differ;Ah Mew;J. Pediatr.,2013

4. Executive functioning profiles from the BRIEF across pediatric medical disorders: age and diagnosis factors;Krivitzky;Child Neuropsychol.,2016

5. Cross-sectional observational study of 208 patients with non-classical urea cycle disorders;Ruegger;J. Inherit. Metab. Dis.,2014

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