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

Aseel Bin SawadArti PothukuchyMark BadeauxVictoria HodsonGillian BubbKristina LindsleyJennifer UyeiGeorge A Diaz

Published in: JIMD reports (2022)

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

Keyphrases

end stage renal disease
newly diagnosed
ejection fraction
chronic kidney disease
prognostic factors
peritoneal dialysis
smoking cessation