I wonder if people truly understand what it means if their rare disease or disorder is on the RARE List™? Last week, the Global Genes Project, leading patient advocacy organizations representing the rare disease community, issued the RARE List™, a stunning 65 page alphabetical listing of roughly 7,000 known rare diseases and disorders. Did you [...]
Why Vote4Hope? How Kickstarting Cures For Rare Disease Through The Pepsi Refresh Project Can Help Kids
Last week, I thought about trying to raise $250k in the Pepsi Refresh Project for The Addi and Cassi Fund and for Niemann Pick Type C disease research but being part of something larger that can make a difference for millions of ‘rare kids’ is important to me. That’s why I decided during the entire [...]
For the past week, I have been obsessing over rare disease statistics ever since it was published that 80% of all rare diseases are ‘ultra orphans’ – affecting 6,000 people per disease state or less – and only 15% of all rare disease drug applications filed with the FDA are for ‘ultra orphans.’ Yesterday, I [...]
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 [...]
Numbers Don’t Add Up – Only 347 New Drugs Approved By FDA For 30 Million Americans With Rare Disease in Past 25 Years?
When it comes to finding Rare Disease statistics, the same old statistics and facts keep popping up again and again. I realize data is difficult to gather on 7000 different rare diseases that afflict 30 million Americans but it’s frustrating that sports teams and Wall Street have more comprehensive statistics when millions of people across [...]