This is a list of the orphan drugs (specialty drugs to treat rare disorders or conditions) approved by the U.S. Food and Drug Administration (FDA) for marketing in the U.S. ... Read More

The U.S. Centers for Disease Control and Prevention (CDC) has identified a Saudi-born man living in Virginia as the third human case of mad cow disease (variant Creutzfeldt-Jakob disease) in ... Read More

Researchers from the Washington University School of Medicine in St. Louis, Missouri, studied data on 291 African American children with sickle cell disease. They wanted to look at the relationship ... Read More

Christmas disease is a form of hemophilia named after the 10-year-old boy with the disease whom researchers studied in 1952 in Oxford, England. Learn about the long history of hemophilia ... Read More

Type II glycogen storage disorder, also known as Pompe disease, is caused by a deficiency of the enzyme GAA. Myozyme (alglucosidase alfa), marketed by Genzyme, replaces the missing enzyme. Researchers ... Read More

VariZIG The U.S. Food and Drug Administration (FDA) granted orphan drug designation on November 7, 2006, to a varicella zoster immune globulin (human) injection called VariZIG, made by Cangene Corporation. VariZIG ... Read More

McCune-Albright syndrome consists of a pattern of bone, endocrine, and skin abnormalities, especially replacement of normal bone with fibrous growth (fibrous dysplasia). Its effects on the body may range from ... Read More

Infants born with neonatal hemochromatosis, an iron storage disorder, often die from it. Liver transplantation, however, may cure the disorder. Researchers at the University Medical Center Hamburg-Eppendorf, Germany, studied 16 ... Read More

Researchers at Uppsala University Hospital in Sweden conducted a population-based study of Swedish hospital patients. The researchers identified 1,636 individuals between the years 1975 and 2001 with Wegener granulomatosis, a ... Read More

In Castleman disease, non-cancerous growths (tumors) develop in lymph node tissue in the body. Three types of Castleman disease have been identified. Little is known about how often the disease ... Read More

Initial tests linked the E. coli outbreak at Taco Bell restaurants in five states to green onions possibly contaminated with a harsh strain of the bacteria called O157:H7. Taco Bell ... Read More

What causes Kawasaki disease, a disorder that creates inflammation of blood vessels throughout the body, is not clear. Some researchers believe it is an infection, others an autoimmune disorder stimulated ... Read More

The National Organization for Rare Disorders (NORD) has launched an online community for people affected by, and concerned about, any of the more than 6,000 rare disorders known to exist. ... Read More

In aplastic anemia, the bone marrow stops making enough red blood cells, white blood cells, and platelets. Aplastic anemia may appear at any age. It may occur as part of ... Read More