On January 30, 2007, the U.S. Food and Drug Administration (FDA) announced a series of initial steps to ensure the safety of drugs before, during, and after approval for marketing. ... Read More

In 2006, researchers at the Johns Hopkins University School of Medicine found that mice with Marfan syndrome, a connective tissue disorder, responded to treatment with losartan (Cozaar), a common blood ... Read More

Loeys-Dietz syndrome is an inherited disorder which affects blood vessels in the body, especially the aorta (the largest artery). It was first described in the medical literature in August 2006. ... Read More

Individuals who have cystic fibrosis, an exocrine disorder, have little pancreatic enzyme produced by their bodies, so they must take enzyme supplements for proper digestion. ALTU-135 (TheraCLEC-Total), produced by Altus ... Read More

Wilms tumor (nephroblastoma) is a kidney cancer that occurs in young children. In 1990, mutations in a gene called WT1 were associated with Wilms tumor, accounting for about 5% of ... Read More

This inherited metabolic disease is one of the lysosomal storage disorders. In Schindler disease, a deficient enzyme (alpha-NAGA) leads to an abnormal accumulation of certain compounds in the cells of ... Read More

Individuals with Rett syndrome, a genetic disorder which mainly affects the brain and nervous system, often have high levels of anxiety and agitation. Researchers from the Baylor College of Medicine ... Read More

Imagine falling asleep while standing in line, driving, or talking, and you have an idea of what narcolepsy, a disorder that causes people to suddenly fall asleep during the day, ... Read More

Fabry disease is a fat storage disorder caused by a deficiency of an enzyme. Since fat doesn't break down properly, part of it accumulates in the blood, blood vessels, and ... Read More

Globoid-cell leukodystrophy, also known as Krabbe disease, is a genetic disorder in which an enzyme (GALC) is deficient. Scientists have tried to replace the defective gene in Krabbe disease through ... Read More

Researchers at the University of Pennsylvania School of Medicine are studying a drug called AEGR-733 for the treatment of familial hypercholesterolemia (FH), an inherited condition in which individuals have very ... Read More

In myelodysplastic syndrome, the bone marrow doesn't make enough normal blood cells (red cells, white cells, and platelets). Although there is no cure for this disorder, there are treatments available, ... Read More

Here are several studies listed at ClinicalTrials.gov that are looking to enroll patients. Some of the studies have been going on for years but are still looking for new individuals ... Read More

Meningitis is an infection which causes inflammation of the membranes covering the brain and spinal cord. It can be caused by a virus (which is the more common kind and ... Read More