Phase Ii Study of an Oral Therapy for Gaucher Disease Yields Positive Results
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Summary
WASHINGTON, May 3 /PRNewswire-USNewswire/ -- Gaucher disease, a rare enzyme deficiency disorder, is one of many conditions with few approved treatment options for patients. In a study published online today in Blood, the journal of the American Society of Hematology, researchers present positive results of a Phase II clinical trial of eliglustat tartrate, an oral therapy in development to treat Gaucher disease.
Gaucher disease is a genetic disorder affecting an estimated 1 in 50,000 to 1 in 100,000 people in the U.S. population. Gaucher disease occurs when a mutation of the glucocerebrosidase gene causes low activity of that enzyme in the body. As a result, harmful fatty substances accumulate in the liver, spleen, bones, and bone marrow, preventing cells and organs from working properly. The primary treatment option is enzyme replacement therapy, which is given intravenously, to break down the accumulated fatty substances.See the full content of this document
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Phase Ii Study of an Oral Therapy for Gaucher Disease Yields Positive Results
Eliglustat tartrate is an oral drug currently in development for Gaucher disease type 1 (GD1). As a substrate reduction therapy, the drug decreases the body's produc...
See the full content of this document
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