Bone involvement is one of the most prevalent aspects of GD and a major cause of pain, disability, and reduced quality of life. All three types of Gaucher disease have bone disease in common and can have serious complications in those affected.
Existing therapies for Gaucher disease (ERT and SRT) are very effective in reversing some of the clinical manifestations of Gaucher disease such as anaemia, thrombocytopenia, etc. but skeletal responses are much slower. Some patients even after many years of treatment, still have bone crisis, bone pain or severe symptoms.
We will have two wonderful speakers:
Each speaker will give a 30 minute lecture, followed by 30 minutes for questions and discussion.