The 2nd Annual Rare Disease Leadership Summit: Financial, Clinical, Marketing and Reimbursement Considerations for Successful Orphan Drug Development, will be held December 5-6, 2007, in Washington, DC. Sponsored by the ... Read More

Although research has shown that lung transplantation adds about 4-5 years to the life of an adult with cystic fibrosis (CF), an exocrine disorder, there is not as much data ... Read More

Mowat-Wilson syndrome consists of a group of birth defects that occur together. It is caused by a genetic defect in chromosome 2. How often Mowat-Wilson syndrome occurs is not known. ... Read More

Aortic dissection, in which the aorta, one of the largest blood vessels in the body, starts to tear, comes on quickly and without warning. What causes the aorta to start ... Read More

Waldenstrom’s macroglobulinemia is a blood cell cancer. White blood cells called B lymphocytes multiply out of control, invading the bone marrow, liver, and spleen. People over age 50 have the ... Read More

On October 29, 2007, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to AVI-4658 (AVI Biopharma, Inc) for the treatment of Duchenne muscular dystrophy, a hereditary progressive ... Read More

Cat eye syndrome, named after how it often affects the appearance of the eyes, is caused by a genetic defect in chromosome 22. There is a wide range of symptoms ... Read More

Lakshmi Tatma, a 2 year old Indian girl, was born with an underdeveloped (parasitic) twin attached to her body, a very rare condition known as isciopagus. The twin had no ... Read More

November 2007 is Pulmonary Hypertension Awareness Month in the U.S. Pulmonary hypertension means increased blood pressure in the blood vessels in the lungs. This can cause shortness of breath, extreme ... Read More

Lymphangiomatosis is a disease in which many tumors grow in the lymph system of the body. Although these tumors are not cancerous, they invade the body tissues and cause pain, ... Read More