New FDA ‘Rare Disease’ Division Being Proposed to U.S. House Committee on Appropriations

The Kakkis Every Life Foundation is leading the effort to request $10 million in the fiscal year 2011 Ag-Rural Development-FDA Appropriations bill directing the U.S. Food and Drug Administration to establish a new review division for Biochemical and Genetic Diseases within the Center for Drug Evaluation Research (CDER), Office of New Drugs. A letter from […]

World Rare Disease Day 2009 Genes Video

I made this video for World Rare Disease Day 2008 to bring global attention to the fact that Rare Disease is not so rare — it can happen to anyone or any family. Although each Rare Disease affects a small number of people, taken all together rare diseases affect millions of people in the world. […]