GlaxoSmithKline (GSK) has announced the formation of a new research unit. The unit will be specialising in the development and commercialisation of medicines for rare diseases. Over 5,500 rare diseases have been identified of which less than 10% are currently being treated, presenting a significant unmet medical need. Despite the rarity of each condition, the number of diseases means that between 6-8% of the population may be affected by a rare disease. Many of these dieses are genetic in origin. They start in childhood and cause lifelong debility and premature death.

During 2009, GSK entered into strategic collaborations with two specialist companies, Prosensa and JCR Pharmaceuticals. The alliance with Prosensa, announced in October 2009, focuses on nucleic acid based therapeutics, correcting gene expression in diseases with large unmet medical needs. The scope of the alliance includes four RNA-based compounds intended to treat specific, but different, subpopulations of patients suffering from Duchenne Muscular Dystrophy (DMD).

As part of the agreement with JCR Pharmaceuticals, a Japanese developer and manufacturer of bioactive products, GSK has obtained global rights to a number of enzyme replacement therapies that could, upon approval, be used to treat orphan diseases such as Hunter syndrome, Fabry disease and Gaucher disease.

GlaxoSmithKline - one of the world's leading research-based pharmaceutical and healthcare companies - is committed to improving the quality of human life by enabling people to do more, feel better and live longer.