Gaucher management guidelines recommend an individualised treatment approach following careful assessment of all disease manifestations due to the heterogeneity of the disease. Pastores G, Weinreb N, Aerts H, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol 2004;41 (suppl 5):4-14 Personalised therapeutic goals and a monitoring strategy that includes quality of life measures are important in effective management of the disease.
The ICGG Gaucher Registry
A program that tracks information about patients' symptoms and experiences in order to improve understanding of Gaucher disease.