Great news for our friends in the the Gaucher community! The FDA and its counterpart across the pond, the European Medicines Agency (EMA), have outlined a new strategy to spur…
Those of us with Gaucher disease who know how much uncertainty, fear, and anger enzyme replacement treatment (ERT) shortages cause, REJOICE! Researchers have found that the oral substrate reduction therapy…
Gaucher disease occurs when the body doesn't produce the enzyme beta-glucocerebrosidase, which is needed to break down fats, or lipids. As a result, fatty deposits collect in the spleen and liver,…
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