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Gaucher disease type 1 management

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Abbreviations: CCL18: chemokine ligand 18; ERT: enzyme replacement therapy; GauSS-I: Gaucher Severity Score Index for type I patients; GD-DS3: Gaucher disease type I severity scoring system; IV: intravenous; Lyso-Gb1/Lyso-GL1: glucosylsphingosine; MDT: multidisciplinary team; NSAIDS: non-steroidal anti-inflammatory drugs; PARC: pulmonary and activation-regulated chemokine; SRT: substrate reduction therapy; SSI: severity score index; TDS: three times daily; Tx: treatment.

(Adapted from: Torralba-Cabeza MÁ, Olivera-González S, Sierra-Monzón JL. The importance of a multidisciplinary approach in the management of a patient with type I Gaucher disease. Diseases 2018;6[3]:69.)

(Source: Louw VJ, Fraser I, Giraldo P. Management goals of type 1 Gaucher disease in South Africa: an expert Delphi consensus document on good clinical practice. PLoS One 2023;18[8]:e0290401. Creative Commons Attribution 4.0 International [CC BY 4.0] license, creativecommons.org/licenses/by/4.0.)