Natural history of arginase 1 deficiency and the unmet needs of patients: A systematic review of case reports-Reference-Cited by-同舟云学术

Natural history of arginase 1 deficiency and the unmet needs of patients: A systematic review of case reports

Published:2022-03-25 Issue:4 Volume:63 Page:330-340
ISSN:2192-8312
Container-title:JIMD Reports
language:en
Short-container-title:JIMD Reports

Author:

Bin Sawad Aseel¹^ORCID,Pothukuchy Arti¹,Badeaux Mark¹,Hodson Victoria¹,Bubb Gillian¹,Lindsley Kristina²,Uyei Jennifer²,Diaz George A.³

Affiliation:

1. Aeglea BioTherapeutics, Inc. Austin Texas USA

2. Health Economics and Outcomes Research ‐ Evidence Synthesis IQVIA, Inc. San Francisco California USA

3. Division of Medical Genetics and Genomics, Department of Genetics and Genomic Sciences Icahn School of Medicine at Mount Sinai New York New York USA

Funder

Aeglea BioTherapeutics

Publisher

Wiley

Subject

Biochemistry, Genetics and Molecular Biology (miscellaneous),Endocrinology, Diabetes and Metabolism,Internal Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jmd2.12283

Reference49 articles.

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4. NORD.National organization for rare disorders. Arginase‐1 Deficiency.2020. Accessed January 20 2021.https://rarediseases.org/rare‐diseases/arginase‐deficiency/#:~:text=Arginase%2D1%20deficiency%20has%20been collectively%20is%20one%20in%2030%2C000.

5. RothK WindleM DescartesM&SteinerR. Arginase deficiency treatment & management. Medscape;2019. Accessed January 25 2021.https://emedicine.medscape.com/article/941838-treatment.

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