Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1

摘要

BackgroundIn 2009, a worldwide supply constraint of imiglucerase led to treatment modifications or interruptions for patients with Gaucher disease (GD) type 1. In France, joint treatment recommendations were issued to protect the most vulnerable patients. This observational study evaluated the impact of imiglucerase treatment modifications on the clinical and biological course of GD.