Notizia - Recent Pharma, Healthcare and Biotech Happenings

Introduction: Any disease that affects less than 200,000 patients or 1 in 1500 is termed as a rare disease. There are around 5000 to 8000 rare disease reported, out which only 10% have cure. According to the European Organization for rare disease, 80% of rare diseases are genetic in nature. Some of the rare diseases include Alzheimer’s disease, Parkinson’s disease, Fabry disease, Gaucher disease, Pompe Disease and Hunter syndrome. Cancers are the most common category of rare diseases. Drugs are often “orphaned”, or never are produced and sold on the market, even when a compound is thought to be useful for the treatment of a rare disease. Figure 1: Rare Cancer Cell A...