CAMBRIDGE, Mass., Feb. 28, 2011 (GLOBE NEWSWIRE) -- Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat severe lipid disorders, announced today its alliance with the National Organization for Rare Disorders (NORD) and observance of the fourth annual Rare Disease Day. NORD is coordinating this observance with the support of more than 200 patient organizations, professional societies, government agencies, medical researchers and pharmaceutical and biotechnology companies to raise awareness about rare diseases.
Aegerion is currently in late-stage clinical development for an orphan drug candidate, lomitapide, a small molecule, once-a-day, oral therapy to treat homozygous familial hypercholesterolemia (HoFH). HoFH is a rare genetic lipid disorder resulting in an accumulation of low-density lipoprotein (LDL-C), often referred to as bad cholesterol, in the blood. Patients with HoFH are at severely high risk of experiencing a heart attack or stroke in their twenties, and if untreated, patients with HoFH generally die before the age of thirty.
A rare disease is one that affects fewer than 200,000 Americans. According to the National Institutes of Health (NIH), there are nearly 7,000 rare diseases affecting nearly 30 million Americans. Rare Disease Day was first observed in Europe in 2008. It was established by EURORDIS, the European Rare Disease Organization. In 2009, EURORDIS partnered with NORD for this initiative in the United States. For more information about Rare Disease Day, visit www.rarediseases.org .Marc D. Beer, CEO of Aegerion, commented, "We share NORD's interest in creating awareness about rare diseases and calling attention to the frequent lack of effective diagnosis and treatment for these underserved patients. It is our ongoing mission to advance research and development and improve care for patients with rare disorders."