The international LOWER (Lomitapide Observational Worldwide Evaluation) treatment registry is now underway and accepting physicians and their eligible patients being treated with lomitapide, according details presented in a poster at the National Lipid Association Scientific Sessions in Chicago.
In an exclusive interview with Rare Disease Report, Don WIlson, MD, FNLA, of Cook Children's Physician Network in Fort Worth, Texas talks about cholesterol and triglyceride testing in pediatric patients.
Deya Corzo, MD, FAAP, FACMG, vice president of US medical affairs at uniQure talks about the clinical trial the company has planned in North America to study the safety and efficacy of Glybera (alipogene tiparvovec) for the treatment of patients with lipoprotein lipase deficiency (also known as familial chylomicronemia syndrome).
Don WIlson, MD, FNLA, of Cook Children's Physician Network in Fort Worth, Texas describes a case of a young boy diagnosed with primary sclerosing cholangitis, an extremely rare autoimmune hepatobiliary disease.
Later this week, the National Lipids Association (NLA) Scientific Sessions will begin in Chicago, Illinois, and will showcase numerous scientific and clinical data on a number of rare lipid conditions.
James Underberg, MD, of NYU Langone Medical Center describes familial chylomicronemia syndrome (FCS), a very rare hereditary condition in which individuals lack properly functioning lipoprotein lipase (LPL), an enzyme that clears triglycerides from plasma.