Presenters: Roscoe O. Brady, MD; Norman W. Barton, MD, PhD; Gregory Grabowski, MD; Gregory Pastores, MD; Henry Mankin, MD
Program covers the natural history of Gaucher disease and the appropriate technology to assess the severity and predict progression. Also current molecular and enzymatic assays for ascertaining affected individuals and carriers, indications for treatment and appropriate modes of therapy, and the goals and consequences of therapeutic interventions will be discussed.
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