Clinical effect and safety profile of pegzilarginase in patients with arginase 1 deficiency-Reference-Cited by-同舟云学术

Clinical effect and safety profile of pegzilarginase in patients with arginase 1 deficiency

Published:2021-01-26 Issue:4 Volume:44 Page:847-856
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:Jrnl of Inher Metab Disea

Author:

Diaz George A.¹^ORCID,Schulze Andreas²,McNutt Markey C.³,Leão‐Teles Elisa⁴,Merritt J. Lawrence⁵,Enns Gregory M.⁶,Batzios Spyros⁷,Bannick Allison⁸,Zori Roberto T.⁹,Sloan Leslie S.¹⁰,Potts Susan L.¹⁰,Bubb Gillian¹⁰,Quinn Anthony G.¹⁰

Affiliation:

1. Icahn School of Medicine at Mount Sinai New York City New York USA

2. University of Toronto and The Hospital for Sick Children Toronto Ontario Canada

3. UT Southwestern Medical Center Dallas Texas USA

4. Centro Hospitalar de São João Porto Portugal

5. University of Washington Seattle Washington USA

6. Stanford University Stanford California USA

7. Great Ormond Street Hospital NHS Trust London UK

8. Children's Hospital of Michigan Wayne State University Detroit Michigan USA

9. University of Florida Gainesville Florida USA

10. Aeglea BioTherapeutics, Inc. Austin Texas USA

Funder

Stanford University

Wayne State University

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jimd.12343

Reference21 articles.

1. Hyperargininemia with Arginase Deficiency

2. Hyperargininemia due to arginase I deficiency: the original patients and their natural history, and a review of the literature

3. Arginase-1 deficiency

4. De DeynP MarescauB QureshiI MoriA.Hyperargininemia: a treatable inborn error of metabolism? The Fourth International Symposium on Guanidino Compounds in Biology and Medicine: Guanidino Compounds in Biology & Medicine II; 1997:53‐69.

5. Amino acids in CSF and plasma in hyperammonaemic coma due to arginase1 deficiency

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