There are more than 6,000 different rare diseases affecting over 60 million people across Europe and North America, alone, and millions more throughout the world. Most of these diseases are genetic, serious, chronic and debilitating. Each disease is different, but they affect people in similar ways. Joining together can help patients and their families find common solutions for care and remind them they are not alone.
As the world marks Rare Disease Day on February 28, 2014, it’s important to remember that patient groups and biotechnology companies are great allies in finding cures and medical breakthroughs for devastating diseases.
At the BIO CEO & Investor Conference in New York, BIO had the opportunity to speak with Hans Schikan, CEO of Prosensa, a company that targets rare diseases – in particular Duchenne Muscular Dystrophy. He highlighted the important role patient advocacy organizations play in his company’s drug development process and the industry’s goal to help save lives and improve quality of life.
Orphan Disease Forum, Tuesday, June 24, 9:00 am – 5:00 pm
Drug development for rare and orphan diseases poses complex challenges to the biopharmaceutical industry, including unique issues around clinical trial recruitment; identification and validation of biomarkers; development of clinical endpoints; and collection of natural history data… Learn more