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

Aseel Bin Sawad, Arti Pothukuchy,Mark Badeaux, Victoria Hodson,Gillian Bubb,Kristina Lindsley,Jennifer Uyei,George A Diaz

JIMD reports（2022）

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摘要

This review illustrates a significant burden of disease and highlights a considerable unmet need for clinically effective treatment options for patients with ARG1-D.

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关键词

ARG1‐D,Arginase 1 deficiency,hyperargininemia,newborn screening,pegzilarginase,systematic literature review,urea cycle disorder

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