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Cerdelga Approved for Gaucher Disease
People with rare disorder lack key enzyme

By Scott Roberts

WEDNESDAY, Aug. 20, 2014 (HealthDay News) -- Cerdelga (eliglustat) has been approved by the U.S. Food and Drug Administration to treat type 1 Gaucher disease, a rare inherited disorder caused by the body's insufficient production of a key enzyme.

Lack of the enzyme glucocerebrosidase causes fatty deposits in the spleen, liver and bone marrow. Symptoms include liver and spleen enlargement, anemia, low blood platelets and bone problems, the FDA said in a news release.

Gaucher affects about 6,000 people in the United States. Cerdelga is a gelatin capsule that's designed to slow the body's production of abnormal fatty deposits.

Cerdelga was evaluated in clinical studies involving 199 people with type 1 Gaucher disease. The most common side effects were fatigue, headache, nausea, diarrhea, and pain of the back, arms and legs, and upper abdomen.

Cerdelga is produced by Genzyme, based in Cambridge, Mass.

More information

The FDA has more about this approval.

Copyright © 2014 HealthDay. All rights reserved. URL:http://consumer.healthday.com/Article.asp?AID=690966

Resources from HONselect: HONselect is the HON's medical search engine. It retrieves scientific articles, images, conferences and web sites on the selected subject.
Liver
Bone and Bones
Blood Platelets
Bone Marrow
Affect
Blood
Nausea
The list of medical terms above are retrieved automatically from the article.

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