Trade Group Study: Hundreds of Rare Disease Drugs in Development

According to a new report released last month by the trade group Pharmaceutical Research and Manufacturers of America (PhRMA), the biopharmaceutical pipeline is innovative and robust, with a high percentage of potential first-in-class medicines (meaning a new treatment where nothing currently exists) targeting diseases with limited treatment options. In addition to identifying medicines in development for conditions and diseases such as septic shock, ovarian cancer, sickle cell disease, and Lou Gehrig’s disease (amyotrophic lateral sclerosis), which haven’t had any new product approvals in the last ten years, the report offers positive news for the rare disease community: one third of the products currently in clinical development have a rare disease designation by the U.S. Food and Drug Administration (FDA).

PhRMA

New ALS Model is Based on Human Cells from Autopsied Tissue

By isolating cells from patients’ spinal tissue within a few days after death, researchers funded by the National Institutes of Health have developed a new model of the paralyzing disease amyotrophic lateral sclerosis (ALS). They found that during the disease, cells called astrocytes become toxic to nerve cells — a result previously found in animal models but not in humans. The new model could be used to investigate many more questions about ALS, also known as Lou Gehrig’s disease.

Neurons

Updated Rates of the Most Common Neurological Disorders

An up-to-date review of the most common neurological disorders in the United States was published in the January 30th issue of Neurology [1]. Researchers reviewed nearly 500 articles published between 1990 and 2005 to determine the rates of prevalence (meaning the total number of cases of a disease in a given population at a specific time; does not convey information about risk) or incidence (meaning the rate of occurrence of new cases of a particular disease in a given population; measures the risk of a disease) for 12 neurological disorders.