- FDA Says First Round of Tests Show No Live Virus in Pasteurized Milk
- King Charles Returns to Duties After Cancer Treatment
- Biden Administration Delays Menthol Cigarette Ban
- Blood Test Might Predict Knee Osteoarthritis Years Early
- Dogs Can Get Lyme Disease, Too
- Spinal Cord Injury Damages Metabolism, and Scientists Now Know Why
- Syphilis Is Increasingly Displaying Atypical, Severe Symptoms
- Climate Change Could Be Good News for Viruses Like COVID
- Vaccines Have Saved 154 Million Lives, Mostly Babies, Over Past 50 Years
- Scientists Discover Cause of Rare Movement Disorder
Cerdelga Approved for Gaucher Disease
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.
Source: HealthDay
Copyright © 2024 HealthDay. All rights reserved.