On August 29, 2007, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to Treanda (bendamustine HCl, developed by Cephalon, Inc.), for the treatment of chronic lymphocytic leukemia ... Read More

September 2007 is National Sickle Cell Disease Awareness Month in the United States. The Sickle Cell Disease Association of America states that it wants the public to reflect on the ... Read More

Takayasu arteritis is a serious chronic inflammatory disease of the large arteries, usually affecting the aorta and its large branches. Although the cause of the disease is unknown, studies suggest ... Read More

A study published in the July 2007 issue of Cancer Epidemiology Biomarkers & Prevention found that obesity, as defined by a body mass index (BMI) of 30 or greater, was ... Read More

Researchers in Cambridge, England, studied the sleeping and waking cycles of mice with the genetic mutation for Huntington disease (HD), an inherited neurological disorder. The researchers found that these mice ... Read More

Newborn babies around the world are tested shortly after birth for genetic or metabolic disorders such as sickle cell disease and phenylketonuria. Early detection, diagnosis, and treatment of these disorders ... Read More

5(S)-(2'-hydroxyethoxy)-20(S)-camptothecin On June 15, 2007, the U.S. Food and Drug Administration (FDA) granted orphan drug status to the drug 5(S)-(2'-hydroxyethoxy)-20(S)-camptothecin (made by ClinTec International, Ltd) for the treatment of osteosarcoma, a ... Read More

Researchers at the National Institute of Allergy and Infectious Diseases and their colleagues studied three youth (ages 10, 11 and 14 years) who had X-linked severe combined immunodeficiency (SCID), in ... Read More

In this disorder, an enzyme (alpha-fucosidase) is missing in the body. Without it, the body can't finish breaking down certain sugar, fat, and protein molecules into their useful parts. The ... Read More

The FACES to FACES 2007 Encephalitis Conference will be held August 17 to 19 in Nashville, Tennessee. FACES (Friends And Caregivers Encephalitis Survivors) welcomes anyone who has been touched by ... Read More

Spinal muscular atrophy is a group of inherited disorders that destroys the nerves controlling voluntary muscle movement. There is no cure, but organizations such as Families of Spinal Muscular Atrophy ... Read More

Eisenmenger syndrome develops in individuals with significant heart defects. In Eisenmenger syndrome, blood pressure increases in the lungs (called pulmonary hypertension). Usually, the syndrome develops while individuals with heart defects ... Read More

Amyotrophic laterals sclerosis (ALS, or Lou Gehrig's disease) is either inherited (about 5 percent of cases) or occurs in a person with no family history of the disease (sporadic form). ... Read More

The U.S. Food and Drug Administration's (FDA) Office of Orphan Products Development (OOPD) is pleased to announce the availability of funds for fiscal year (FY) 2009 and FY 2010 grant ... Read More