Overcoming the Challenges of Rare Disease Drug Development

The translation of a drug from discovery to development faces a number of obstacles, but these can be amplified for rare disease therapies in part because of the smaller populations, heterogeneous nature, and often poorly understood development of a disease. The Catalyst program at the Clinical & Translational Science Institute at the University of California, San Francisco will be holding a one-day symposium on March 3 with leading researchers, drug developers, and patient advocates about the translational challenges rare disease therapies face and how to address them. We spoke to Cathy Tralau-Stewart, interim director of UCSF's Catalyst Program, about the upcoming symposium, the unique translational challenges rare disease therapies face, and what can be done to overcome them. To view the symposium agenda or to register, you can paste this URL (http://bit.ly/2lWmYbB) into your browser.